5-HT1D receptor

FINTEPLA is approved by the U.S. Food and Drug Administration (FDA) for the treatment of seizures associated with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) in patients two years of age and older1 Results showed the most common treatment-emergent adverse events were pyrexia, nasopharyngitis and decreased appetite, with no valvular heart disease or pulmonary arterial hypertension observed in this study2 FINTEPLA was associated with a statistically significant and clinically meaningful sustained reduction in monthly convulsive seizure frequency (MCSF)2,3 ATLANTA, March 12, 2025 /PRNewswire/ -- UCB, a global biopharmaceutical company, today announced that Epilepsia published findings from the final analysis of the long-term open-label extension (OLE) study evaluating the safety and effectiveness of FINTEPLA® (fenfluramine) in patients with Dravet syndrome (DS). Results showed that FINTEPLA was generally well tolerated. The most common treatment-emergent adverse events (TEAEs) were pyrexia, nasopharyngitis and decreased appetite.2 The efficacy analysis showed a statistically significant and clinically meaningful sustained reduction in monthly convulsive seizure frequency (MCSF) with FINTEPLA in both children and adults with DS.2 FINTEPLA® (fenfluramine) is approved by the U.S. Food and Drug Administration (FDA) for the treatment of seizures associated with Dravet syndrome and Lennox-Gastaut syndrome in patients two years of age and older. (PRNewsfoto/UCB) "Anti-seizure medications that provide long-term tolerability and effectiveness are needed for patients with DS, who often have many other medical conditions that are adversely impacted by seizure frequency and severity," said Ingrid E. Scheffer, MBBS, Ph.D., co-author of the publication and laureate professor, chair of pediatric neurology at the University of Melbourne. "This analysis, showing that FINTEPLA was generally well tolerated over the long-term and increased seizure-free days, builds on previous findings from randomized, controlled trials in adults and children with DS and provides additional evidence of the sustained tolerability and efficacy of this treatment." The OLE study enrolled patients with DS who had participated in one of three randomized, placebo-controlled Phase 3 trials of FINTEPLA or who were 19 to 35 years old and had not received FINTEPLA prior to study entry. The primary objective of the OLE study was to assess the long-term safety and tolerability of FINTEPLA. Efficacy endpoints evaluated the median percentage change in MCSF from Day 1 to end of study (EOS) and Day 31 to EOS compared with baseline; the proportion of patients to achieve seizure reduction thresholds; the longest convulsive seizure-free interval; and the percentage of convulsive seizure-free days in the modified intent-to-treat (mITT) population. Clinical Global Impression-Improvement (CGI-I) ratings by caregiver and investigator and changes in baseline in the Quality of Life Childhood Epilepsy (QOLCE) score were also evaluated. Results showed that FINTEPLA was generally well tolerated with only 11 patients (2.9%) discontinuing therapy due to an adverse event. TEAEs occurring in ≥10% of patients were pyrexia, nasopharyngitis, decreased appetite, seizure, decreased blood glucose, diarrhea, abnormal echocardiogram (only physiologic regurgitation), upper respiratory tract infection, influenza, vomiting, and ear infection. No valvular heart disease or pulmonary arterial hypertension was observed in this study. Efficacy outcomes showed a sustained reduction in MCSF with FINTEPLA in the mITT population of 324 patients, with a median change from Day 1 to EOS compared with baseline of -66.8% (p<0.001) and -67.8% (p<0.001) from Day 31 to EOS compared with baseline. FINTEPLA was effective in both patients less than six years old and those six years or older. The analysis showed that 64.2% of patients experienced a 50% or more reduction in MCSF; two patients were seizure-free throughout the OLE. From Month 1 to EOS, there was a significant 20.3% median increase in convulsive seizure-free days (p<0.0001). Caregivers rated 61.3% of patients as much/very much improved at the last visit on the CGI-I, as did 62.6% of investigators. Significant improvements from baseline were observed in several subscales of the QOLCE, including energy/fatigue, language, general health, QOL and overall QOL. DS is a rare, severe, drug-resistant developmental and epileptic encephalopathy (DEE) that is characterized by seizure onset before the first year of life and developmental slowing or regression.4,5 DS is often challenging to diagnose.6 Patients often experience multiple seizure types5 and effects beyond seizures, including cognitive impairment, speech impairment, sleep and gait issues, and behavioral issues, including impulsivity and hyperactivity, that can adversely impact quality of life.5-7 "The ability to meaningfully address the complexities of Dravet, inclusive of the severe and often debilitating seizures, is critical for patients living with this rare syndrome and those who care for them," said Brad Chapman, head of U.S. epilepsy and rare syndromes at UCB. "The publication of these new long-term findings, in which patients were exposed to FINTEPLA for up to 3.5 years and experienced durable and clinically meaningful reductions in seizure frequency, emphasizes the benefit of this treatment and our commitment to helping improve outcomes for patients with DS and other rare epilepsies." About FINTEPLA Important Safety Information about FINTEPLA® (fenfluramine) in the US INDICATIONS AND USAGE FINTEPLA is indicated for the treatment of seizures associated with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) in patients 2 years of age and older. IMPORTANT SAFETY INFORMATION BOXED WARNING: VALVULAR HEART DISEASE and PULMONARY ARTERIAL HYPERTENSION There is an association between serotonergic drugs with 5-HT2B receptor agonist activity, including fenfluramine (the active ingredient in FINTEPLA), and valvular heart disease and pulmonary arterial hypertension. Echocardiogram assessments are required before, during, and after treatment with FINTEPLA. FINTEPLA is available only through a restricted program called the FINTEPLA REMS. CONTRAINDICATIONS FINTEPLA is contraindicated in patients with hypersensitivity to fenfluramine or any of the excipients in FINTEPLA and with concomitant use, or within 14 days of the administration, of monoamine oxidase inhibitors because of an increased risk of serotonin syndrome. WARNINGS AND PRECAUTIONS Valvular Heart Disease and Pulmonary Arterial Hypertension (see Boxed Warning): Because of the association between serotonergic drugs with 5-HT2B receptor agonist activity, including fenfluramine (the active ingredient in FINTEPLA), and valvular heart disease (VHD) and pulmonary arterial hypertension (PAH), cardiac monitoring via echocardiogram is required prior to starting treatment, during treatment, and after treatment with FINTEPLA concludes. Cardiac monitoring via echocardiogram can aid in early detection of these conditions. In clinical trials for DS and LGS of up to 3 years in duration, no patient receiving FINTEPLA developed VHD or PAH. Monitoring: Prior to starting treatment, patients must undergo an echocardiogram to evaluate for VHD and PAH. Echocardiograms should be repeated every 6 months, and once at 3-6 months post treatment with FINTEPLA. The prescriber must consider the benefits versus the risks of initiating or continuing treatment with FINTEPLA if any of the following signs are observed via echocardiogram: valvular abnormality or new abnormality; VHD indicated by mild or greater aortic regurgitation or moderate or greater mitral regurgitation, with additional characteristics of VHD (eg, valve thickening or restrictive valve motion); PAH indicated by elevated right heart/pulmonary artery pressure (PASP >35 mmHg). FINTEPLA REMS Program (see Boxed Warning): FINTEPLA is available only through a restricted distribution program called the FINTEPLA Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribers must be certified by enrolling in the FINTEPLA REMS. Prescribers must counsel patients receiving FINTEPLA about the risk of VHD and PAH, how to recognize signs and symptoms of VHD and PAH, the need for baseline (pretreatment) and periodic cardiac monitoring via echocardiogram during FINTEPLA treatment, and cardiac monitoring after FINTEPLA treatment. Patients must enroll in the FINTEPLA REMS and comply with ongoing monitoring requirements. The pharmacy must be certified by enrolling in the FINTEPLA REMS and must only dispense to patients who are authorized to receive FINTEPLA. Wholesalers and distributors must only distribute to certified pharmacies. Further information is available at or by telephone at 1-877-964-3649. Decreased Appetite and Decreased Weight: FINTEPLA can cause decreases in appetite and weight. Decreases in weight appear to be dose related. Approximately half of the patients with LGS and most patients with DS resumed the expected measured increases in weight during the open-label extension studies. Weight should be monitored regularly during treatment with FINTEPLA, and dose modifications should be considered if a decrease in weight is observed. Somnolence, Sedation, and Lethargy: FINTEPLA can cause somnolence, sedation, and lethargy. Other central nervous system (CNS) depressants, including alcohol, could potentiate these effects of FINTEPLA. Prescribers should monitor patients for somnolence and sedation and should advise patients not to drive or operate machinery until they have gained sufficient experience on FINTEPLA to gauge whether it adversely affects their ability to drive or operate machinery. Suicidal Behavior and Ideation: Antiepileptic drugs (AEDs), including FINTEPLA, increase the risk of suicidal thoughts or behaviors in patients taking these drugs for any indication. Patients treated with an AED for any indication should be monitored for the emergence or worsening of depression, suicidal thoughts or behaviors, or any unusual changes in mood or behavior. Anyone considering prescribing FINTEPLA or any other AED must balance the risk of suicidal thoughts or behaviors with the risks of untreated illness. Epilepsy and many other illnesses for which AEDs are prescribed are themselves associated with morbidity and mortality and an increased risk of suicidal thoughts and behaviors. Should suicidal thoughts and behaviors emerge during treatment, consider whether the emergence of these symptoms in any given patient may be related to the illness being treated. Withdrawal of Antiepileptic Drugs: As with most AEDs, FINTEPLA should generally be withdrawn gradually because of the risk of increased seizure frequency and status epilepticus. If withdrawal is needed because of a serious adverse reaction, rapid discontinuation can be considered. Serotonin Syndrome: Serotonin syndrome, a potentially life-threatening condition, may occur with FINTEPLA, particularly during concomitant administration of FINTEPLA with other serotonergic drugs, including, but not limited to, selective serotonin-norepinephrine reuptake inhibitors (SNRIs), selective serotonin reuptake inhibitors (SSRIs), tricyclic antidepressants (TCAs), bupropion, triptans, dietary supplements (eg, St. John's Wort, tryptophan), drugs that impair metabolism of serotonin (including monoamine oxidase inhibitors [MAOIs], which are contraindicated with FINTEPLA), dextromethorphan, lithium, tramadol, and antipsychotics with serotonergic agonist activity. Patients should be monitored for the emergence of signs and symptoms of serotonin syndrome, which include mental status changes (eg, agitation, hallucinations, coma), autonomic instability (eg, tachycardia, labile blood pressure, hyperthermia), neuromuscular signs (eg, hyperreflexia, incoordination), and/or gastrointestinal symptoms (eg, nausea, vomiting, diarrhea). If serotonin syndrome is suspected, treatment with FINTEPLA should be stopped immediately and symptomatic treatment should be started. Increase in Blood Pressure: FINTEPLA can cause an increase in blood pressure. Rare cases of significant elevation in blood pressure, including hypertensive crisis, has been reported in adult patients treated with fenfluramine, including patients without a history of hypertension. In clinical trials for DS and LGS of up to 3 years in duration, no pediatric or adult patient receiving FINTEPLA developed hypertensive crisis. Monitor blood pressure in patients treated with FINTEPLA. Glaucoma: Fenfluramine can cause mydriasis and can precipitate angle closure glaucoma. Consider discontinuing treatment with FINTEPLA in patients with acute decreases in visual acuity or ocular pain. ADVERSE REACTIONS The most common adverse reactions observed in DS studies (incidence at least 10% and greater than placebo) were decreased appetite; somnolence, sedation, lethargy; diarrhea; constipation; abnormal echocardiogram; fatigue, malaise, asthenia; ataxia, balance disorder, gait disturbance; blood pressure increased; drooling, salivary hypersecretion; pyrexia; upper respiratory tract infection; vomiting; decreased weight; fall; status epilepticus. The most common adverse reactions observed in the LGS study (incidence at least 10% and greater than placebo) were diarrhea; decreased appetite; fatigue; somnolence; vomiting. DRUG INTERACTIONS Strong CYP1A2, CYP2B6, or CYP3A Inducers: Coadministration with strong CYP1A2, CYP2B6, or CYP3A inducers will decrease fenfluramine plasma concentrations. If coadministration of a strong CYP1A2, CYP2B6, or CYP3A inducer with FINTEPLA is necessary, monitor the patient for reduced efficacy and consider increasing the dosage of FINTEPLA as needed. If a strong CYP1A2, CYP2B6, or CYP3A inducer is discontinued during maintenance treatment with FINTEPLA, consider gradual reduction in the FINTEPLA dosage to the dose administered prior to initiating the inducer. Strong CYP1A2 or CYP2D6 Inhibitors: Coadministration with strong CYP1A2 or CYP2D6 inhibitors will increase fenfluramine plasma concentrations. If FINTEPLA is coadministered with strong CYP1A2 or CYP2D6 inhibitors, the maximum daily dosage of FINTEPLA is 20 mg. If a strong CYP1A2 or CYP2D6 inhibitor is discontinued during maintenance treatment with FINTEPLA, consider gradual increase in the FINTEPLA dosage to the dose recommended without CYP1A2 or CYP2D6 inhibitors. If FINTEPLA is coadministered with stiripentol and a strong CYP1A2 or CYP2D6 inhibitor, the maximum daily dosage of FINTEPLA is 17 mg. USE IN SPECIFIC POPULATIONS In patients with severe impairment of kidney function (estimated glomerular filtration rate [eGFR]) 15 to 29 mL/min/1.73m2, dosage adjustments are recommended. FINTEPLA has not been studied in patients with kidney failure (eGFR <15 mL/min/1.73m2). Combined molar exposures of fenfluramine and norfenfluramine were increased in subjects with various degrees of hepatic impairment (Child-Pugh Class A, B, and C), necessitating a dosage adjustment in these patients. To report SUSPECTED ADVERSE REACTIONS, contact UCB, Inc. at 1 844-599-2273 or FDA at 1-800-FDA-1088 or . Please see full Prescribing Information , including Boxed Warning and Medication Guide , for additional Important Safety Information on FINTEPLA. For further information, contact UCB: Investor Relations Antje Witte T: +32.2.559.94.14 email [email protected] US Communications Becky Malone T +1.919.605.9600 Email [email protected] About UCB UCB, Brussels, Belgium () is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With approximately 9,000 people in approximately 40 countries, the company generated revenue of €5.3 billion in 2023. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCBUSA. About Epilepsy Epilepsy is a common neurological condition worldwide and affects approximately 50 million people. Epilepsy can develop in any person at any age and is usually diagnosed after a person has had at least two seizures (or after one seizure with a high risk for more) that were not caused by some known medical condition. About UCB in Epilepsy For three decades we've been committed to people living with epilepsy and their families, surrounding the patient and caregiver through every step of their care journey. At our core we have a responsibility to elevate the healthcare ecosystem. Because of this, we have established our legitimacy on the front lines of epilepsy research, development, and treatment innovation. But our past breakthroughs are only a prologue to our future. We will continue to reimagine how we care for patients, leveraging today's expertise for a better tomorrow. With so much experience behind us and so much potential ahead, we are more invested than ever in profoundly improving the lives of those living with or caring for those with epilepsy or a rare epilepsy syndrome – through our relentless pursuit of a seizure-free life. Forward looking statements This press release may contain forward-looking statements including, without limitation, statements containing the words "believes", "anticipates", "expects", "intends", "plans", "seeks", "estimates", "may", "will", "continue" and similar expressions. These forward-looking statements are based on current plans, estimates and beliefs of management. All statements, other than statements of historical facts, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, arbitration, political, regulatory or clinical results or practices and other such estimates and results. 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Important factors that could result in such differences include: changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms or within expected timing, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, product liability claims, challenges to patent protection for products or product candidates, competition from other products including biosimilars, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in tax laws or the administration of such laws, and hiring and retention of its employees. There is no guarantee that new product candidates will be discovered or identified in the pipeline, will progress to product approval or that new indications for existing products will be developed and approved. Movement from concept to commercial product is uncertain; preclinical results do not guarantee safety and efficacy of product candidates in humans. So far, the complexity of the human body cannot be reproduced in computer models, cell culture systems or animal models. The length of the timing to complete clinical trials and to get regulatory approval for product marketing has varied in the past and UCB expects similar unpredictability going forward. Products or potential products, which are the subject of partnerships, joint ventures or licensing collaborations may be subject to differences disputes between the partners or may prove to be not as safe, effective or commercially successful as UCB may have believed at the start of such partnership. UCB's efforts to acquire other products or companies and to integrate the operations of such acquired companies may not be as successful as UCB may have believed at the moment of acquisition. Also, UCB or others could discover safety, side effects or manufacturing problems with its products and/or devices after they are marketed. The discovery of significant problems with a product similar to one of UCB's products that implicate an entire class of products may have a material adverse effect on sales of the entire class of affected products. Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment, including pricing pressure, political and public scrutiny, customer and prescriber patterns or practices, and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement activities and outcomes. Finally, a breakdown, cyberattack or information security breach could compromise the confidentiality, integrity and availability of UCB's data and systems. Given these uncertainties, you should not place undue reliance on any of such forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labelling in any market, or at any particular time, nor can there be any guarantee that such products will be or will continue to be commercially successful in the future. UCB is providing this information, including forward-looking statements, only as of the date of this press release. UCB expressly disclaims any duty to update any information contained in this press release, either to confirm the actual results or to report or reflect any change in its forward-looking statements with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless such statement is required pursuant to applicable laws and regulations. Additionally, information contained in this document shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any offer, solicitation or sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such jurisdiction. References FINTEPLA (fenfluramine) oral solution: U.S. prescribing information. Smyrna, GA: UCB, Inc. Scheffer IE, Nabbout R, Lagae L, et al. Long-term safety and effectiveness of fenfluramine in children and adults with Dravet syndrome. Epilepsia. 2025. Scheffer IE, Nabbout R, Lagae L, et al. Safety and Effectiveness of Adjunctive Fenfluramine in an Open-Label Extension Study of Patients With Dravet Syndrome. [Poster] Presented at: International Child Neurology Conference; 6-10 May 2024; Cape Town, South Africa; 667. Dravet C. The core Dravet syndrome phenotype. Epilepsia. 2011;52(Suppl. 2):3-9. Nabbout R, Hyland K, Loftus R, et al. Dravet syndrome seizure frequency and clustering: Placebo-treated patients in clinical trials. Epilepsy Behav. 2024;155:109774. doi: 10.1016/j.yebeh.2024.109774. Lo Barco T, Kuchenbuch M, Garcelon N, Neuraz A, Nabbout R. Improving early diagnosis of rare diseases using Natural Language Processing in unstructured medical records: an illustration from Dravet syndrome. Orphanet J Rare Dis. 2021;16(309). . Strzelczyk A, Schubert-Bast S. Psychobehavioural and cognitive adverse events of anti-seizure medications for the treatment of developmental and epileptic encephalopathies. CNS Drugs. 2022;36(10):1079-1111. doi: 10.1007/s40263-022-00955-9. US-FA-2500169 Date of preparation: February 2025 FINTEPLA® is a registered trademark of the UCB Group of Companies. ©2025 UCB, Inc., Smyrna, GA 30080. All rights reserved. SOURCE UCB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

SYMBRAVO demonstrated statistically significantly greater migraine treatment response compared to prior treatment with an oral CGRP inhibitor (p<0.001, mTOQ-4 total score, primary endpoint) 2-hour pain freedom for most attacks reported at least half the time by 47.9% of patients after SYMBRAVO versus 1.0% after oral CGRPs (p<0.001) 24-hour or greater sustained pain relief after a single dose reported by 47.9% of patients after SYMBRAVO versus 16.7% after oral CGRPs (p<0.001) Ability to quickly return to normal activities reported by 51.0% of patients after SYMBRAVO versus 11.5% after oral CGRPs (p<0.001) SYMBRAVO improved quality of life compared to after oral CGRPs (p<0.001, MSQ) NEW YORK, Feb. 24, 2025 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company leading a new era in the treatment of central nervous system (CNS) disorders, today announced that the EMERGE Phase 3 trial of SYMBRAVO® (MoSEIC™ meloxicam and rizatriptan) in patients experiencing inadequate response to oral CGRP inhibitors met its primary endpoint, with SYMBRAVO demonstrating statistically significantly greater migraine treatment response compared to oral CGRP inhibitors, as measured by the Migraine Treatment Optimization Questionnaire (mTOQ-4). SYMBRAVO is a novel multi-mechanistic approach to treating migraine that targets multiple pathways underlying a migraine attack. In the trial, SYMBRAVO rapidly and substantially improved migraine pain and most bothersome symptoms. EMERGE was an open-label trial that enrolled migraine patients who were undergoing treatment with an oral CGRP inhibitor for at least one month and experiencing an inadequate response to the oral CGRP inhibitor, with treatment response assessed using the mTOQ-4. Enrolled patients were switched to treatment with SYMBRAVO for their next four attacks. Treatment responses after the oral CGRP inhibitor treatment period and after the SYMBRAVO treatment period were compared. A total of 96 patients were enrolled and 365 migraine attacks were treated with SYMBRAVO in the trial. EMERGE met the primary endpoint by demonstrating statistically significantly greater migraine treatment response with SYMBRAVO compared to treatment response with prior oral CGRP inhibitors, as assessed by the mTOQ-4 total score (5.2 versus 2.8, p<0.001). Statistically significantly greater proportions of patients achieved clinical response on the 2-hour pain freedom, sustained pain freedom, ability to return to normal activities, and ability to plan daily activities mTOQ-4 items with SYMBRAVO compared to oral CGRP inhibitors: Pain freedom within 2 hours for most attacks was reported half the time or more by 47.9% of patients after treatment with SYMBRAVO, compared to 1.0% of patients after treatment with oral CGRPs (p<0.001). Sustained relief of migraine pain for at least 24 hours following a single dose of medication was reported half the time or more by 47.9% of patients after treatment with SYMBRAVO, compared to 16.7% of patients after treatment with oral CGRPs (p<0.001). The ability to quickly return to normal activities after taking their medication was reported half the time or more by 51.0% of patients after treatment with SYMBRAVO, compared to 11.5% of patients after treatment with oral CGRPs (p<0.001). The proportion of patients who reported being comfortable enough with their medication to be able to plan daily activities half the time or more was 63.5% after treatment with SYMBRAVO, compared to 26.0% after treatment with oral CGRPs (p<0.001). Further, SYMBRAVO treatment resulted in a statistically significant improvement in overall quality of life and daily functioning, as assessed by all three domains of the Migraine-Specific Quality of Life Questionnaire (MSQ), compared to after treatment with oral CGRP inhibitors (p=0.003 to <0.001). Richard B. Lipton, MD, Professor of Neurology and Director of the Montefiore Headache Center, Albert Einstein College of Medicine, commented, “The results of the EMERGE study demonstrate significant improvements in migraine treatment response with SYMBRAVO for patients previously experiencing inadequate response to oral CGRPs based on the mTOQ-4. Migraine is a disabling neurological condition, and the multiple mechanisms of action of SYMBRAVO may be relevant to the complex and heterogenous nature of this serious condition. These data from the EMERGE study are compelling and provide further evidence for the utility of SYMBRAVO across a variety of migraine settings.” In this population of patients with a prior inadequate response to an oral CGRP inhibitor, SYMBRAVO rapidly and substantially relieved migraine pain within 2 hours, with benefits sustained through 24 and 48 hours after a single dose. Across all treated attacks, pain relief 2 hours after dosing with SYMBRAVO was achieved by 50.0% of patients, with some patients experiencing pain relief as early as 30 minutes after dosing. The pain relief achieved 2 hours after dosing was sustained through 24 and 48 hours by 78% and 75% of patients respectively. Pain freedom and freedom from the most bothersome symptom 2 hours after dosing with SYMBRAVO was achieved by 22.5% and 26.6% of patients, respectively. Overall improvement of migraine, measured using the Patient Global Impression of Change (PGI-C), was experienced early and in a substantial proportion of patients after treatment with SYMBRAVO. Overall improvement of their migraine was reported by 26.0% of patients 30 minutes post dose, and by 69.2% of patients 2 hours post dose. SYMBRAVO was well tolerated with a safety profile that was consistent with what has been previously observed in prior studies. The most commonly reported adverse events (≥2%) were fatigue (3.1%), nausea (3.1%), vomiting (2.1%), muscle tightness (2.1%), and dizziness (2.1%). Herriot Tabuteau, MD, Chief Executive Officer of Axsome Therapeutics, said, “We’re pleased to share the results of the Phase 3 EMERGE trial, which further underscore the robust efficacy of SYMBRAVO and its potential to effectively treat migraine attacks across a range of patient populations with varying pain intensities and prior responses to acute treatments. We look forward to launching SYMBRAVO in the U.S. in the coming months and offering a new treatment option that could make a meaningful difference for patients suffering from this disabling condition.” About the EMERGE Trial EMERGE (Evaluating Outcomes of AXS-07 after Acute Gepant Failures) was a Phase 3, open-label, multicenter trial to evaluate the efficacy and safety of SYMBRAVO in the acute treatment of migraine in patients experiencing inadequate response to an oral calcitonin gene-related peptide (CGRP) inhibitor. Eligible patients must have been using an oral CGRP inhibitor for the acute treatment of migraine for at least 1 month prior to enrollment (having treated at least 4 migraines with an oral CGRP inhibitor) and have had an inadequate response to the oral CGRP inhibitor. An inadequate response was defined as a score of ≤7 on the Migraine Treatment Optimization Questionnaire (mTOQ-4), including a score of 1 (“less than half the time”) or 0 (“rarely” or “never”) on Question 2 (achievement of pain freedom 2 hours after taking migraine medication). Enrolled patients were switched from their oral CGRP inhibitor to SYMBRAVO and treated the next 4 migraine attacks with SYMBRAVO over a period of up to 8 weeks. A total of 96 patients were enrolled in the trial. The primary efficacy endpoint to assess treatment response with SYMBRAVO versus oral CGRP inhibitors was the change in the mTOQ-4 total score from the oral CGRP inhibitor treatment period to the SYMBRAVO treatment period. About the Migraine Treatment Optimization Questionnaire (mTOQ-4) The mTOQ-4 is a validated questionnaire that assesses the adequacy of migraine treatment efficacy based on four aspects of response to acute treatment: 2-hour pain freedom; sustained pain freedom; ability to quickly return to daily activities; and comfort planning daily activities. Each of the 4 items is scored, using frequency-based options, as never [0], rarely [0], less than half the time [1], and half of the time or more [2]. Total scores range from 0 to 8 with higher total scores corresponding to greater treatment optimization. A total score of 8 corresponds to maximum treatment efficacy, with total scores of 4-7 corresponding to moderate, 1-3 to poor, and 0 to very poor treatment optimization.1 About Migraine Migraine is a serious neurological condition characterized by recurrent attacks of pulsating, often severe and disabling head pain associated with nausea, sensitivity to light, and sensitivity to sound.2 It is estimated that over 39 million Americans suffer from migraine, and it is the leading cause of disability among neurological disorders in the United States according to the American Migraine Foundation.3-5 Extensive surveys of migraine sufferers underscore the unmet need for therapies that work faster, more consistently, and result in less symptom recurrence.6,7 Over 70% of patients report experiencing an inadequate response to their oral, acute migraine treatment.8 About SYMBRAVO SYMBRAVO is a novel, oral, single-dose medicine approved for the acute treatment of migraine with or without aura in adults. SYMBRAVO consists of MoSEIC™ meloxicam and rizatriptan. Meloxicam is a new molecular entity for migraine enabled by Axsome’s MoSEIC (Molecular Solubility Enhanced Inclusion Complex) technology, which enables the rapid absorption of meloxicam while maintaining a long plasma half-life. Meloxicam is a COX-2 preferential non-steroidal anti-inflammatory drug (NSAID) and rizatriptan is a 5-HT1B/1D agonist. SYMBRAVO is designed to provide rapid, enhanced, and consistent migraine pain relief, and reduced symptom recurrence. The exact mechanism of action of SYMBRAVO in the treatment of acute migraine is unknown. For more information, visit www.symbravo.com. What is SYMBRAVO (sim-BRAH-voh)? SYMBRAVO is a combination of meloxicam (an NSAID) and rizatriptan (a triptan). SYMBRAVO is an oral prescription medicine used to treat acute migraine headaches with or without aura in adults. SYMBRAVO is not used to prevent or decrease the number of migraine headaches you have or for treatment of hemiplegic or basilar migraines. SYMBRAVO is not indicated as a treatment for cluster headaches or for use in children. WHAT IS THE MOST IMPORTANT INFORMATION I SHOULD KNOW ABOUT SYMBRAVO? SYMBRAVO may increase the risk of a heart attack or stroke that can lead to death. This risk may happen early in treatment and may increase with increasing doses, and longer use, of NSAIDs. Do not take SYMBRAVO right before or after a heart surgery called a “coronary artery bypass graft” (CABG). Avoid taking SYMBRAVO after a recent heart attack unless your healthcare provider (HCP) tells you to. You may have an increased risk of another heart attack if you take NSAIDs after a recent heart attack. Stop taking SYMBRAVO and get emergency help right away if you have any of the following symptoms which can be indicative of a heart attack or stroke: discomfort in your chest that lasts for more than a few minutes, or that goes away and comes back severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw pain or discomfort in your arms, back, neck, jaw, or stomach shortness of breath with or without chest discomfort breaking out in a cold sweat nausea or vomiting feeling lightheaded weakness in one part or one side of your body slurred speech People with risk factors for heart disease should not take SYMBRAVO unless a heart exam is done and shows no problem. You have higher risk for heart disease if you: have high blood pressure have high cholesterol smoke are overweight have diabetes or a family history of diabetes SYMBRAVO can increase the risk of potentially life-threatening bleeding, ulcers, and tears (perforation) of the esophagus (tube leading from the mouth to the stomach), stomach, and intestines that can occur anytime during use and without warning symptoms. SYMBRAVO may cause serious allergic or skin reactions which can be life-threatening. Stop taking SYMBRAVO and get emergency help right away if you develop: sudden wheezing or problems breathing or swallowing rash or reddening of your skin with blisters or peeling blisters or bleeding of your lips, eye lids, mouth, nose, or genitals swelling of your lips, tongue, throat or body fainting SYMBRAVO already contains an NSAID (meloxicam). Do not use SYMBRAVO with other medicines to lessen pain or fever or with other medicines for colds or sleeping problems without talking to your HCP first, because they may contain an NSAID also. Do not take SYMBRAVO if you: have or had heart problems or right before or after heart bypass surgery have or had a stroke or transient ischemic attack (TIA) have or had blood vessel problems of your legs and arms, stomach (ischemic bowel disease), or kidneys have or had hemiplegic or basilar migraines have uncontrolled high blood pressure take propranolol containing medicines have taken other triptan or ergot-containing medicines within the last 24 hours take an antidepressant medicine called monoamine oxidase inhibitor (MAOI) or have taken a MAOI within the last 2 weeks are allergic to meloxicam, rizatriptan, NSAIDs, or any of the ingredients in SYMBRAVO have had an asthma attack, hives, or other allergic reaction after taking aspirin or any other NSAIDs have moderate to severe kidney problems and are at risk of kidney failure or if you are on dialysis SYMBRAVO may cause serious side effects. These serious side effects include: heartbeats that are too fast or too slow (arrhythmias) new or worse high blood pressure heart failure life-threatening skin reactions liver or kidney problems including organ failure low red blood cell count (anemia) asthma attacks in people who have asthma Medication Overuse Headaches: Some people who use too many SYMBRAVO tablets may have worse headaches. If your headaches get worse, your HCP may decide to stop your treatment with SYMBRAVO. Stop taking SYMBRAVO and get emergency help right away if you have any of the following: Stomach and intestinal problems. Symptoms of gastrointestinal and colonic ischemic events may include sudden or severe stomach pains even after meals; sudden weight loss; severe nausea, vomiting, constipation, diarrhea; and bloody diarrhea. Circulation problems to legs and feet. Symptoms of peripheral vascular ischemia may include cramping and pain in your legs and hips; heaviness or tightness in leg muscles; burning, aching, numbness, tingling, or weakness in your legs, feet, or toes; cold feelings or color changes in one or both legs or feet. Serotonin syndrome. Can happen when taking SYMBRAVO with antidepressant medicines called SSRIs or SNRIs. Stop taking SYMBRAVO and call your doctor right away if you have any of the following symptoms: mental status changes including agitation, hallucinations, or coma fast heartbeat changes in your blood pressure increased body temperature tight muscles trouble walking mental status changes including agitation, hallucinations, or coma fast heartbeat changes in your blood pressure increased body temperature tight muscles trouble walking Stop taking SYMBRAVO and call your healthcare provider right away if you have any of the following symptoms: nausea vomiting blood more tired or weaker than usual blood in your bowel movement or it is black and sticky like tar diarrhea itching, skin rash, or blisters with fever unusual weight gain your skin or eyes look yellow indigestion or stomach pain swelling of the arms, legs, hands, or feet flu-like symptoms tenderness in your right upper side vision problems COMMON SIDE EFFECTS The most common side effects of SYMBRAVO include dizziness and tiredness. These are not all the possible side effects of SYMBRAVO. Tell your doctor if you have any side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1–800-FDA-1088. BEFORE USING Tell your HCP about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. It is important to tell your HCP if you are taking: Propranolol containing medicines such as Inderal® LA or Innopran® XL Aspirin or other anti-coagulants (blood thinners) Medicines to help your mood including SSRIs and SNRIs If you are unsure if you take any of these medicines, ask your HCP. They can tell you if it is safe to take SYMBRAVO with your other medicines. Tell your HCP if you are pregnant or plan to become pregnant. SYMBRAVO is not recommended during pregnancy. Taking NSAIDs, including SYMBRAVO, at about 20 weeks of pregnancy or later may harm your unborn baby. NSAIDs, including SYMBRAVO, should not be taken after about 30 weeks of pregnancy. Tell your HCP if you are breastfeeding or plan to breastfeed. Propranolol containing medicines such as Inderal® LA or Innopran® XL Aspirin or other anti-coagulants (blood thinners) Medicines to help your mood including SSRIs and SNRIs Tell your HCP about all your medical conditions, including if you: have or have had heart problems, high blood pressure, chest pain, or shortness of breath have any risk factors for heart or blood vessel problems have kidney or liver problems have asthma Review the list below with your HCP. SYMBRAVO may not be right for you if: take daily preventative aspirin you are pregnant or plan to become pregnant you are breastfeeding or plan to breastfeed HOW TO TAKE SYMBRAVO is available by prescription only. Take SYMBRAVO exactly as instructed by your HCP. The maximum daily dose of SYMBRAVO is 1 tablet. Talk to your HCP about what to do if your headache does not go away or comes back. Take SYMBRAVO for the shortest time needed. Swallow SYMBRAVO tablets whole. Do not crush, chew, or divide the tablets. SYMBRAVO can be taken with or without food. Do not give SYMBRAVO to other people. If you take too much SYMBRAVO call your poison control center at 1-800-222-1222 or go to the nearest hospital emergency room right away. LEARN MORE For more information about SYMBRAVO, call 866-496-2976 or visit SYMBRAVO.com. This summary provides basic information about SYMBRAVO but does not include all information known about this medicine. Read the information that comes with your prescription each time your prescription is filled. This information does not take the place of talking with your doctor. Be sure to talk to your doctor or other HCP about SYMBRAVO and how to take it. Your HCP is the best person to help you decide if SYMBRAVO is right for you. SYM CON BS 01/2025 Please see full Prescribing Information, including Boxed Warning for risk of serious cardiovascular and gastrointestinal adverse events, and Medication Guide. About Axsome Therapeutics Axsome Therapeutics is a biopharmaceutical company leading a new era in the treatment of central nervous system (CNS) conditions. We deliver scientific breakthroughs by identifying critical gaps in care and develop differentiated products with a focus on novel mechanisms of action that enable meaningful advancements in patient outcomes. Our industry-leading neuroscience portfolio includes FDA-approved treatments for major depressive disorder, excessive daytime sleepiness associated with narcolepsy and obstructive sleep apnea, and migraine, and multiple late-stage development programs addressing a broad range of serious neurological and psychiatric conditions that impact over 150 million people in the United States. Together, we are on a mission to solve some of the brain’s biggest problems so patients and their loved ones can flourish. For more information, please visit the Company’s website at www.axsome.com. Forward Looking Statements Certain matters discussed in this press release are “forward-looking statements”. The Company may, in some cases, use terms such as “predicts,” “believes,” “potential,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. In particular, the Company’s statements regarding trends and potential future results are examples of such forward-looking statements. The forward-looking statements include risks and uncertainties, including, but not limited to, the commercial success of the Company’s Sunosi®, Auvelity®, and Symbravo® products and the success of the Company’s efforts to obtain any additional indication(s) with respect to solriamfetol and/or AXS-05; the Company’s ability to maintain and expand payer coverage; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund the Company’s disclosed clinical trials, which assumes no material changes to the Company’s currently projected revenues or expenses), futility analyses and receipt of interim results, which are not necessarily indicative of the final results of the Company’s ongoing clinical trials, and/or data readouts, and the number or type of studies or nature of results necessary to support the filing of a new drug application (“NDA”) for any of the Company’s current product candidates; the Company’s ability to fund additional clinical trials to continue the advancement of the Company’s product candidates; the timing of and the Company’s ability to obtain and maintain U.S. Food and Drug Administration (“FDA”) or other regulatory authority approval of, or other action with respect to, the Company’s product candidates, including statements regarding the timing of any NDA submission; the Company’s ability to successfully defend its intellectual property or obtain the necessary licenses at a cost acceptable to the Company, if at all; the successful implementation of the Company’s research and development programs and collaborations; the success of the Company’s license agreements; the acceptance by the market of the Company’s products and product candidates, if approved; the Company’s anticipated capital requirements, including the amount of capital required for the commercialization of Sunosi, Auvelity, and Symbravo and for the Company’s commercial launch of its other product candidates, if approved, and the potential impact on the Company’s anticipated cash runway; the Company’s ability to convert sales to recognized revenue and maintain a favorable gross to net sales; unforeseen circumstances or other disruptions to normal business operations arising from or related to domestic political climate, geo-political conflicts or a global pandemic and other factors, including general economic conditions and regulatory developments, not within the Company’s control. The factors discussed herein could cause actual results and developments to be materially different from those expressed in or implied by such statements. The forward-looking statements are made only as of the date of this press release and the Company undertakes no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstance. Investors: Mark Jacobson Chief Operating Officer (212) 332-3243 mjacobson@axsome.com Media: Darren Opland Director, Corporate Communications (929) 837-1065 dopland@axsome.com References Source: Axsome Therapeutics, Inc.

In this article, we present a comprehensive overview of drugs that received their first global approvals in January 2025, along with an analysis of these therapies. The drugs discussed in this article include small molecules, biologics, and combination drugs that were globally approved for the first time in January 2025. It is important to note that this compilation excludes generic drugs, biosimilars, traditional Chinese medicines, and allergen extracts. For combination drugs, only Type 4 drugs approved by the FDA are included. Additionally, drugs that were first approved as new drugs by the FDA in January 2025 but had previously received approval in other countries are also included in this analysis and are marked with an asterisk (*) for clarity. Which drugs were approved by the FDA?1. Whole Blood and Blood Components Whole Blood and Blood Components, specifically convalescent plasma derived from COVID-19 patients, received approval from the U.S. Food and Drug Administration (FDA) on January 3, 2025. The approval allows the use of high-titer SARS-CoV-2 antibody-containing whole blood-derived convalescent plasma and apheresis-derived convalescent plasma as a treatment for COVID-19 infection. Developed and manufactured by OneBlood, Inc., COVID-19 convalescent plasma is human plasma collected from eligible blood donors by FDA-registered or licensed blood establishments. These donors must have recovered from symptomatic SARS-CoV-2 infection within the past six months and exhibit high levels of anti-SARS-CoV-2 antibodies in their plasma. The plasma’s neutralizing activity is determined based on donor antibody levels assessed through laboratory testing. Specifically, donor samples are evaluated using the GenScript cPass surrogate virus neutralization assay, which measures the inhibition of wild-type receptor-binding domain (RBD) binding to ACE2. The required inhibition rate must be equal to or greater than 80%. This product is indicated for the treatment of COVID-19 in immunocompromised or immunosuppressed patients, particularly those at high risk of severe disease progression in both inpatient and outpatient settings. Convalescent plasma may be contraindicated in patients with a history of severe allergic reactions to plasma transfusion. The generally recommended dose is one unit (approximately 200 mL), with adjustments based on the patient's clinical response and physician’s discretion. During administration, ABO blood type compatibility must be ensured, but Rh(D) compatibility is not required. Additionally, precautions should be taken to prevent container breakage or thawing issues during storage, and standard transfusion safety protocols should be followed to mitigate potential adverse effects and risks. 2. Datopotamab Deruxtecan* Datopotamab deruxtecan is a Trop-2-targeting antibody-drug conjugate (ADC) jointly developed by Daiichi Sankyo and AstraZeneca. On December 27, 2024, Japan approved datopotamab deruxtecan for the treatment of adult patients with unresectable or metastatic hormone receptor-positive (HR+)/HER2-negative breast cancer who have received prior chemotherapy. This marked the drug’s first global marketing authorization. Subsequently, on January 17, 2025, the FDA also approved datopotamab deruxtecan for the treatment of unresectable or metastatic, previously treated HR+/HER2-negative breast cancer in adults. This approval was supported by data from the Phase III TROPION-Breast01 trial, which demonstrated a significant improvement in progression-free survival (PFS) and a reduction in the risk of disease progression or death compared to standard therapy. Datopotamab deruxtecan is designed to precisely target cancer cells expressing the Trop-2 antigen. Trop-2 is a protein widely expressed in various solid tumors, particularly breast and lung cancers. The drug consists of a humanized IgG1 monoclonal antibody conjugated to the potent topoisomerase I inhibitor deruxtecan (DXd). The antibody specifically binds to the Trop-2 antigen, while DXd functions as a cytotoxic chemotherapy agent that is released inside tumor cells, disrupting DNA replication and inducing cancer cell death. These components are linked by a cleavable tetrapeptide linker, ensuring stability in circulation and facilitating targeted drug release within tumor cells, thereby maximizing therapeutic efficacy. Clinical trial results demonstrated that datopotamab deruxtecan provides significant efficacy in treating HR+/HER2-negative advanced breast cancer. In the pivotal Phase III TROPION-Breast01 study, the drug outperformed the standard therapy, docetaxel, in prolonging progression-free survival. With a median follow-up of 10.8 months, the median PFS in the datopotamab deruxtecan group was 6.9 months, compared to 4.9 months in the docetaxel group (HR = 0.63, 95% CI: 0.52–0.76, P < 0.0001), representing a 37% reduction in the risk of disease progression or death. Additionally, datopotamab deruxtecan demonstrated a high objective response rate (ORR), further reinforcing its potential as a novel cancer therapy. The drug also exhibited a favorable safety profile, with a lower incidence of grade ≥3 treatment-related adverse events compared to conventional chemotherapy, contributing to improved patient quality of life. 3. Meloxicam/Rizatriptan The combination drug of meloxicam and rizatriptan, marketed under the brand name Symbravo, received approval from the FDA on January 30, 2025, for the treatment of migraines. This innovative small-molecule drug integrates two distinct mechanisms of action to address migraine symptoms effectively. Developed by Axsome Therapeutics, Inc., meloxicam is a selective cyclooxygenase-2 (COX-2) inhibitor primarily used for pain and inflammation relief. Its high selectivity for COX-2 over COX-1 helps minimize the common gastrointestinal side effects associated with traditional nonsteroidal anti-inflammatory drugs (NSAIDs). By inhibiting the COX-2 enzyme, meloxicam reduces prostaglandin production, thereby alleviating pain. Rizatriptan, on the other hand, is a serotonin 5-HT1D and 5-HT1B receptor agonist, commonly used for the acute treatment of migraine attacks. It works by constricting dilated cerebral blood vessels and preventing the release of calcitonin gene-related peptide (CGRP) from nerve terminals—two key mechanisms believed to be crucial for migraine relief. Additionally, research suggests that rizatriptan may exert effects on the central nervous system, modulating specific receptors involved in pain signal transmission, further enhancing its analgesic efficacy. The uniqueness of AXS-07 lies in its combination of these two active ingredients, aiming to leverage both the anti-inflammatory and analgesic properties of meloxicam and the migraine-specific physiological effects of rizatriptan. This dual-action approach provides a comprehensive treatment strategy, targeting both the pain and inflammation associated with migraines while addressing additional migraine-related symptoms. As a result, Symbravo offers patients more effective relief compared to traditional monotherapies. 4. Suzetrigine Suzetrigine received approval from the FDA on January 30, 2025, as a small-molecule drug for the treatment of moderate to severe acute pain in adults. The drug's uniqueness lies in its selective targeting of the voltage-gated sodium ion channel Nav1.8, a channel specifically expressed in peripheral nociceptive neurons responsible for transmitting pain signals. Developed by Vertex Pharmaceuticals, Inc., Suzetrigine is an X-type sodium channel α-subunit blocker that selectively inhibits Nav1.8 to prevent pain signal transmission. This high selectivity allows Suzetrigine to effectively relieve pain without affecting other types of sodium channels, thereby reducing the risk of side effects. Notably, Suzetrigine's mechanism avoids the addictive potential and adverse effects commonly associated with opioid analgesics, such as respiratory depression and constipation. This makes it a crucial new option for patients seeking non-opioid pain management solutions. In the United States, over 80 million people require medication annually for managing moderate to severe acute pain, highlighting the urgent need for effective and non-addictive pain relief options. The successful development and approval of Suzetrigine mark the first market entry of a pain medication with a novel mechanism in nearly two decades. Clinical trials have demonstrated that Suzetrigine significantly reduced pain scores in patients undergoing abdominoplasty and bunionectomy, with no observed risk of addiction. Suzetrigine represents a breakthrough in acute pain management, offering patients an innovative non-opioid, highly targeted analgesic option. Which drugs were approved by the NMPA?1. Amimestrocel Amimestrocel is a human umbilical cord-derived mesenchymal stem cell (MSC) injection developed by Bosheng Excellence Biotechnology. On January 2, 2025, it received conditional approval from China’s National Medical Products Administration (NMPA) through the priority review and approval process, making it the first stem cell therapy to be approved for marketing in China. Amimestrocel is indicated for the treatment of steroid-refractory acute graft-versus-host disease (aGVHD) with predominant gastrointestinal involvement in patients aged 14 and older. Graft-versus-host disease (GVHD) is a severe complication that may occur after allogeneic hematopoietic stem cell transplantation when donor immune cells recognize the recipient’s body as "foreign" and launch an immune attack. A multicenter, randomized, double-blind, parallel, placebo-controlled Phase II clinical trial demonstrated that, in an intent-to-treat analysis, the MSC group exhibited superior therapeutic efficacy compared to the placebo group on Day 28. Additionally, clinical trial data showed that the therapeutic effects of Amimestrocel injection gradually became evident within an average of two weeks, with significant benefits observed particularly in patients with intestinal involvement. Amimestrocel injection has shown promising efficacy in treating steroid-refractory aGVHD. According to clinical trial data, the treatment demonstrated a gradual therapeutic effect at a median of two weeks. Patients who completed eight infusions, especially those with gastrointestinal involvement, derived significant benefits from MSC therapy. These findings suggest that Amimestrocel may be particularly suitable for patients who have not responded well to conventional second-line treatments. 2. Recaticimab Recaticimab received approval from the NMPA on January 8, 2025, marking its official entry into the Chinese market. Developed by Hengrui Medicine, Recaticimab is a humanized monoclonal antibody specifically targeting proprotein convertase subtilisin/kexin type 9 (PCSK9). PCSK9 primarily influences cholesterol metabolism by promoting the degradation of low-density lipoprotein receptors (LDLRs). Under normal conditions, LDLRs are located on the surface of hepatocytes, where they capture and remove low-density lipoprotein cholesterol (LDL-C) from the bloodstream. However, when PCSK9 binds to LDLRs, it triggers their internalization and subsequent degradation in lysosomes, thereby reducing the liver’s ability to clear LDL-C. Recaticimab specifically binds to PCSK9, preventing its interaction with LDLRs, thereby preserving LDLRs from degradation. This leads to an increased number of LDLRs on the hepatocyte surface, enhancing the body’s ability to clear LDL-C. Recaticimab not only increases the number of LDLRs but also enhances their function. Since LDLRs play a key role in reducing serum LDL-C levels, increasing their quantity and activity is crucial for managing dyslipidemia. Additionally, Recaticimab utilizes "YTE" amino acid mutation technology to optimize its molecular structure, extending its half-life to approximately 27 days, significantly longer than other similar drugs. This allows for longer dosing intervals, such as every 4 or 8 weeks, improving patient adherence by reducing the inconvenience of frequent injections. This extended duration provides a more convenient option for the long-term management of hypercholesterolemia. From a clinical perspective, Phase III clinical trial results demonstrate that Recaticimab significantly reduces LDL-C, total cholesterol (TC), non-high-density lipoprotein cholesterol (non-HDL-C), apolipoprotein B (ApoB), and lipoprotein(a) [Lp(a)], whether used as an adjunct to statins or as monotherapy. Moreover, it exhibits an excellent safety profile. Recaticimab is particularly suitable for patients who have poor responses to statins or are statin-intolerant, including those with primary hypercholesterolemia (both heterozygous familial and non-familial) and mixed dyslipidemia. The approval of Recaticimab brings new hope to the field of cardiovascular disease, offering significant benefits in improving patient quality of life and preventing cardiovascular events. 3. Prusogliptin Prusogliptin received approval from the NMPA in China on January 8, 2025. Developed by CSPC Ouyi Pharmaceutical, Prusogliptin is a novel oral dipeptidyl peptidase-IV (DPP-4) inhibitor designed for the treatment of type 2 diabetes in adults. Its primary mechanism of action involves selectively and potently inhibiting DPP-4, thereby preventing the enzymatic degradation of glucagon-like peptide-1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP). These two incretin hormones stimulate pancreatic β-cells to release insulin in response to food intake while simultaneously reducing α-cell secretion of glucagon, helping to regulate blood glucose levels. By inhibiting DPP-4, Prusogliptin prolongs the activity of GLP-1 and GIP, promoting postprandial insulin secretion, enhancing β-cell glucose sensitivity, and reducing unnecessary glucagon secretion, ultimately aiding in maintaining blood glucose levels within a healthier range. Prusogliptin plays a crucial role in increasing the levels of endogenous GLP-1 and GIP. Under normal physiological conditions, these incretin hormones function as "intelligent" regulators of blood glucose, promoting insulin secretion only when glucose levels are elevated while avoiding excess insulin release under normal or low glucose conditions, thereby minimizing the risk of hypoglycemia. Moreover, unlike traditional insulin secretagogues that directly stimulate insulin secretion, Prusogliptin indirectly enhances endogenous GLP-1 function, making it less likely to cause hypoglycemia or lead to weight gain—important advantages for the long-term management of type 2 diabetes. A study led by Peking University First Hospital demonstrated that patients in the Prusogliptin group experienced a significant reduction in glycated hemoglobin (HbA1c) levels compared to the placebo group, confirming its strong glucose-lowering efficacy. Additionally, Prusogliptin is suitable for monotherapy or combination therapy with metformin, making it particularly beneficial for patients who respond poorly to or cannot tolerate metformin. It is indicated for improving glycemic control in adult patients with type 2 diabetes, either as monotherapy or in combination with metformin. Furthermore, Prusogliptin exhibits sustained glucose-lowering effects, a low risk of hypoglycemia, and does not contribute to weight gain. It also has a favorable safety profile, with a low incidence of adverse reactions and minimal drug interactions. Notably, for patients with mild to moderate renal impairment, Prusogliptin does not require dose adjustment, further expanding its eligible patient population. 4. Senaparib Senaparib was approved for market authorization in China on January 14, 2025. As a small-molecule chemical drug, Senaparib primarily targets poly (ADP-ribose) polymerase 1 and 2 (PARP1 and PARP2), exerting its anticancer effects by inhibiting the activity of these enzymes. Developed by IMPACT Therapeutics, Senaparib operates based on the principle of synthetic lethality, playing a crucial role in the repair of single-strand DNA breaks. When these enzymes are inhibited, cancer cells with BRCA mutations or other homologous recombination repair deficiencies (HRD) are less able to effectively repair DNA damage, making them more prone to cell death. As a result, Senaparib is particularly suited for tumors that rely on PARP-mediated DNA repair pathways for survival. According to the NMPA approval, Senaparib is indicated in China for the maintenance treatment of adult patients with advanced epithelial ovarian cancer, fallopian tube cancer, or primary peritoneal cancer following first-line platinum-based chemotherapy. This means that for patients who have achieved complete or partial remission after receiving first-line platinum-based chemotherapy, Senaparib provides a new maintenance treatment option aimed at prolonging progression-free survival and potentially improving overall survival outcomes. The approval of Senaparib is based on the results of the FLAMES study, a randomized, double-blind, placebo-controlled, multicenter Phase III clinical trial. The results demonstrated that, compared to placebo, Senaparib significantly prolonged median progression-free survival (mPFS). Additionally, regardless of BRCA mutation status, patients derived benefits from the treatment. The drug also exhibited good tolerability and a manageable safety profile. 5. Limertinib Limertinib is a novel small-molecule chemical drug that received marketing approval from the NMPA on January 14, 2025. It is a specifically designed inhibitor targeting the epidermal growth factor receptor (EGFR) T790M mutation. As a third-generation EGFR tyrosine kinase inhibitor (EGFR-TKI), Limertinib selectively targets and inhibits EGFR proteins carrying the T790M mutation. This mutation commonly occurs in non-small cell lung cancer (NSCLC) patients who develop resistance after treatment with first- or second-generation EGFR-TKIs. By selectively binding to the EGFR T790M mutation site, Limertinib effectively blocks signal transduction pathways, thereby inhibiting tumor cell proliferation and spread. In a pivotal Phase IIB clinical study, Limertinib demonstrated significant antitumor activity. A total of 301 patients with locally advanced or metastatic NSCLC who had progressed after prior EGFR-TKI therapy and tested positive for the EGFR T790M mutation participated in the study. The results, assessed by an independent review committee (IRC), showed an objective response rate (ORR) of 68.8%, a disease control rate (DCR) of 92.4%, a median duration of response (DoR) of 11.1 months, and a median progression-free survival (PFS) of 11.0 months. Furthermore, for patients with evaluable intracranial lesions, Limertinib also demonstrated promising efficacy, with an IRC-assessed best ORR of 65.9% and a median PFS of 10.6 months, indicating its potential effectiveness against central nervous system (CNS) metastases. Limertinib is indicated for the treatment of adult patients with locally advanced or metastatic NSCLC who have progressed after prior EGFR-TKI therapy and have been confirmed to harbor the EGFR T790M mutation. This indication addresses a critical unmet need in treating resistance caused by specific genetic mutations, offering new hope to affected patient populations. 6. Pegylated Recombinant Human Granulocyte Colony-Stimulating Factor Pegylated recombinant human granulocyte colony-stimulating factor (PEG-rhG-CSF) was approved for marketing in China on January 14, 2025, for the prevention and treatment of chemotherapy-induced neutropenia. Developed by Hangzhou Jiuyuan Gene Engineering, PEG-rhG-CSF is a long-acting biologic drug designed to address chemotherapy-induced neutropenia. This drug is a conjugate of recombinant human granulocyte colony-stimulating factor (rhG-CSF) and polyethylene glycol (PEG), which extends the drug’s half-life in vivo and reduces the frequency of administration. As a CSF-3R (colony-stimulating factor 3 receptor) agonist, PEG-rhG-CSF activates CSF-3R to stimulate the proliferation and differentiation of hematopoietic stem cells and progenitor cells in the bone marrow, leading to an increase in circulating neutrophils and enhancing the body's ability to resist infections. Compared to conventional rhG-CSF, PEG-rhG-CSF offers significant advantages, including prolonged duration of action and reduced injection frequency, which are crucial for improving patient adherence and quality of life. Additionally, the development of this drug addresses the clinical need for efficient, safe, and easy-to-manage treatment options. 7. Efsubaglutide Alfa Efsubaglutide Alfa is an innovative fusion protein drug that was first approved by the NMPA of China on January 24, 2025. Developed by Guangzhou Yinnuo Pharmaceutical, Efsubaglutide Alfa is a glucagon-like peptide-1 receptor agonist (GLP-1RA). It is engineered as a recombinant protein by conjugating the GLP-1 receptor agonist to the Fc fragment of human immunoglobulin G2 (IgG2). This allows it to enhance insulin secretion in a glucose-dependent manner while inhibiting glucagon release, effectively controlling blood glucose levels in adult patients with type 2 diabetes. What sets Efsubaglutide Alfa apart is its ultra-long-acting profile. The drug exhibits higher affinity for the GLP-1 receptor and has a prolonged degradation time in the body, with a half-life of 204 hours. This extended half-life enables a dosing regimen of once weekly or even once every two weeks, significantly improving patient convenience and treatment adherence. Additionally, clinical studies have shown that Efsubaglutide Alfa not only significantly lowers blood glucose levels but also improves lipid profiles, promotes weight loss, and offers potential cardiovascular protective effects. This makes it a comprehensive metabolic management option for patients with type 2 diabetes. Which drugs were approved by the TGA?Garadacimab Garadacimab was first approved in Australia on January 24, 2025, as a novel treatment for hereditary angioedema (HAE). Developed by CSL Behring LLC, Garadacimab is a fully human IgG4 monoclonal antibody specifically designed to target and inhibit activated Factor XII (FXIIa), a key protein in the pathological process of HAE. By targeting and inhibiting FXIIa, the drug prevents excessive activation of the kallikrein-kinin system, thereby reducing the production of bradykinin, which is responsible for HAE symptoms. This mechanism of action provides patients with a long-acting and highly specific prophylactic treatment, significantly reducing the frequency of disease attacks and improving patients' quality of life. Clinical studies, including the VANGUARD trial, have demonstrated that long-term prophylactic treatment with Garadacimab effectively controls acute HAE attacks. Compared to the placebo group, patients receiving Garadacimab reported a higher quality-of-life rating, indicating not only its medical superiority but also its positive impact on patients' overall well-being. Additionally, the drug is administered via subcutaneous injection once every 30 days, greatly enhancing treatment adherence and convenience for patients. The approval of Garadacimab marks a significant advancement in HAE treatment, particularly in the field of prophylactic therapy. Unlike traditional C1 esterase inhibitor replacement therapy or direct blockade of the bradykinin B2 receptor, Garadacimab achieves therapeutic effects by modulating the upstream kallikrein-kinin pathway. This breakthrough not only provides HAE patients with a new treatment option but also showcases the potential of biotechnology in developing targeted therapies for specific molecular pathways. How to obtain the latest research advancements in the field of biopharmaceuticals? In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!