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Last update 26 May 2025

Digoxin

Last update 26 May 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

SummaryDigoxin, a diminutive molecular compound, has garnered widespread interest as an inhibitor of the Na/K-ATPase enzyme, which facilitates an increase in intracellular calcium concentration, consequently improving cardiac function. Although primarily targeted for the treatment of heart failure, atrial fibrillation, and assorted cardiac arrhythmias, Digoxin's effectiveness and low-cost nature has garnered it significant acclaim since its first approval by GSK Plc in 1954, and continues to be prescribed frequently despite its age. However, caution must be exercised due to its slender therapeutic window and propensity for toxicity, necessitating frequent and thorough monitoring of patients taking Digoxin by their healthcare provider.

Drug Type

Small molecule drug

Synonyms

12β-hydroxydigitoxin, Digaoxin, Digoxin (JP17/USP)

+ [8]

Target

Na/K-ATPase

Action

inhibitors

Mechanism

Na/K-ATPase inhibitors(Sodium/potassium-transporting ATPase inhibitors)

Therapeutic Areas

Cardiovascular Diseases

Active Indication

Arrhythmias, CardiacAcute congestive heart failureTachycardia+ [2]

Inactive Indication

Chemotherapy-induced nausea and vomitingDiabetes Mellitus, Type 2Epilepsy+ [2]

Originator Organization

GSK Plc

Active Organization

Covis Pharma Holdings BVHikma Pharmaceuticals International Ltd.Taiyo Pharma Co., Ltd.

+ [2]

Inactive Organization

GSK Plc

Tesaro, Inc.

Theracos, Inc.

+ [1]

License Organization-

Drug Highest PhaseApproved

First Approval Date

United States (16 Nov 1954),

Atrial FibrillationHeart Failure

Regulation-

Structure/Sequence

Molecular FormulaC41H64O14

InChIKeyLTMHDMANZUZIPE-PUGKRICDSA-N

CAS Registry20830-75-5

226

Clinical Trials associated with Digoxin

NCT06240403

/ Not yet recruitingPhase 2IIT

Digoxin: a New Senolytic to Repair Dysfunctional Adipose Tissue in Patients With Heart Failure and Type II Diabetes Mellitus

In pilot studies the investigators have shown that subcutaneous adipose tissue (SAT) from patients with reduced ejection fraction heart failure (HFrEF) and type 2 diabetes mellitus (T2DM) is dysfunctional. Endothelial cells from the adipose tissue from these patients are senescent and have deleterious effects on healthy human subcutaneous adipocytes, including increasing expression of IL-6 (gene and protein) and reducing glucose uptake. Digoxin, a well-established treatment for HFrEF, selectively clears these senescent endothelial cells and prevents adipocyte dysfunction. This study will examine the effect of digoxin on adipose tissue on the burden of senescent cells.

Start Date01 Sep 2025

Sponsor / Collaborator

University of Leeds

NCT06701812

/ RecruitingPhase 2IIT

Evaluation of Digoxin for Relapsed Non-WNT, Non-SHH Medulloblastoma

The purpose of this study is to evaluate the efficacy of digoxin in treating relapsed non-SHH, non-WNT medulloblastoma in pediatric and young adult patients.

Start Date01 May 2025

Sponsor / Collaborator

H. Lee Moffitt Cancer Center & Research Institute, Inc.

NCT06588699

/ Not yet recruitingPhase 2IIT

Clinical Trial of Oral Digoxin In NASH (CODIN)

Nonalcoholic steatohepatitis (NASH) is a severe subtype of nonalcoholic fatty liver disease (NAFLD) which affects 1 in 3 Americans. The mainstay of treatment for NASH, which was recently renamed metabolic associated steatohepatitis (MASH), involves lifestyle interventions to promote weight loss and to treat comorbidities such as hypertension, hyperlipidemia, and diabetes mellitus. There is thus, a substantial unmet need for pharmacological therapies that are effective for treatment of NASH, especially in those with fibrosis which is the main predictor of disease progression and mortality among NASH patients. The repurposing of presently available drugs would help expedite the search for agents effective in treating NASH. The cardiac glycoside digoxin is currently used in the management of heart failure and supraventricular tachyarrhythmias. The investigators and other groups have demonstrated that digoxin protects the liver from various forms of acute and chronic liver injury. The investigators preliminary data in healthy human subject indicate an immunomodulatory effect of low dose oral digoxin with no adverse side effects. This study proposes to demonstrate the clinical benefits of digoxin on NASH and on liver fibrosis, thus supporting the repurposing of digoxin as treatment for NASH.

Start Date30 Apr 2025

Sponsor / Collaborator

Yale University

[+1]

100 Clinical Results associated with Digoxin

100 Translational Medicine associated with Digoxin

100 Patents (Medical) associated with Digoxin

18,096

Literatures (Medical) associated with Digoxin

31 Dec 2025·Journal of Maternal-Fetal & Neonatal Medicine

Stress hyperglycemia ratio: a novel predictor of left ventricular dysfunction in peripartum cardiomyopathy

Article

Author: Liu, Baoli ; Liu, Wenxian ; Chen, Liying ; Zhu, Jiajia

OBJECTIVE:

This study aims to assess the predictive value of the stress hyperglycemia ratio (SHR) for left ventricular (LV) systolic dysfunction in patients with peripartum cardiomyopathy (PPCM).

METHODS:

We conducted a retrospective analysis of 78 consecutive PPCM patients from January 2007 to March 2023. Their clinical, laboratory, and auxiliary examination data were collected. The estimated average glucose (eAG) was calculated using the formula: eAG = [1.59 × hemoglobin A1c (%) -%2.59]. The SHR was determined by the formula: SHR = (blood glucose at admission)/eAG. The primary outcome measured was the recovery of LV systolic function. A receiver operating characteristic (ROC) curve was used to evaluate the SHR. Logistic regression analysis was performed to identify risk factors for LV systolic dysfunction in PPCM patients.

RESULTS:

The mean random blood glucose level in the PPCM patients was 6.38 mmol/L, with an SHR of 1.16. Among these patients, 37 (47.4%) exhibited persistent LV systolic dysfunction during follow-up. The SHR was significantly higher in the non-recovery group than in the recovery group (1.45 vs. 0.91, p < .001). An SHR cutoff of 1.079 predicted persistent LV systolic dysfunction with a sensitivity of 81.1% and a specificity of 90.2%, yielding a Youden index of 0.713. Logistic regression identified an SHR ≥ 1.079, a left ventricular end-diastolic diameter (LVEDD) > 55 mm, and digoxin usage as risk factors for LV systolic dysfunction.

CONCLUSIONS:

PPCM patients with an SHR of 1.079 or higher should receive increased scrutiny for persistent LV systolic dysfunction.

31 Dec 2025·SCANDINAVIAN CARDIOVASCULAR JOURNAL

The use of antiarrhythmic drugs for atrial fibrillation in Finland 2007–2018

Article

Author: Marjamaa, Annukka ; Siponen, Rasmus ; Lehto, Mika ; Hartikainen, Juha ; Teppo, Konsta ; Putaala, Jukka ; Airaksinen, Juhani ; Aro, Aapo ; Mustonen, Pirjo ; Salmela, Birgitta ; Haukka, Jari ; Virrankorpi, Janne ; Linna, Miika ; Lappalainen, Antti ; Halminen, Olli

BACKGROUND:

Patients with atrial fibrillation (AF) are often treated with antiarrhythmic drugs (AADs) to maintain sinus rhythm and with heart rate-lowering drugs to achieve the optimal rate control. In this study, we investigated trends in the use of AADs and rate control drugs in Finnish patients with AF.

METHODS AND RESULTS:

The Finnish AntiCoagulation in Atrial Fibrillation (FinACAF) study is a nationwide study including all patients with AF in Finland from 2007 to 2018. The number of AAD purchases and the proportions of all prevalent AF patients in a certain year of interest were calculated. In total, 391030 AF patients were identified between 2007 and 2018, and 39,816 (10.2%) of them had purchased either class I or III AADs. The proportion of patients using classes I and III AADs decreased from 8.6% to 6.3%. Flecainide and amiodarone were the most often used AADs. The use of flecainide and amiodarone decreased from 4.9% to 3.9% and 1.9% to 1.5%, respectively. The proportion of patients on beta-blockers remained stable at 75%. Dronedarone became available in 2011 when it also was the most used (0.8% of patients), but the use decreased thereafter. The use of sotalol and digoxin decreased from 1.5% to 0.6% and 24.6% to 11.0% over the study period.

CONCLUSION:

The number of AAD purchases increased alongside with the increasing prevalence of AF, whereas the proportion of AF patients on classes I and III AADs and digoxin decreased between 2007 and 2018. Flecainide remained the most used AAD followed by amiodarone.

01 Aug 2025·FOOD CHEMISTRY

Investigation into the effects of acute heat stress on stress level, meat quality, myofibrillar proteins properties and serum metabolites release of Muscovy ducks (Cairina moschata)

Article

Author: Huang, Tianran ; Xiao, Mengchao ; Huang, Jichao ; Li, Zimu ; Zhu, Zongshuai ; Huang, Ming

This study systematically investigated the effects of acute heat stress (AHS) on Muscovy ducks, focusing on plasma stress indicators, meat quality, myofibrillar proteins (MPs) properties, and serum metabolite profiles. AHS significantly elevated stress level, impairing meat quality, as evidenced by higher L* value, shear force, drip loss, and cooking loss (P < 0.05). It also induced MPs oxidation and aggregation, reflected by elevated carbonyl content, turbidity, zeta potential, and particle size. MPs structural alterations were confirmed by fluorescence quenching and increased exposure of hydrophobic groups. Correlation analysis revealed strong associations between physicochemical changes and MPs oxidation. Metabolomic analysis identified 161 and 105 differential metabolites in the CON vs. LS and CON vs. SS comparisons, respectively, involving 25 metabolic pathways related to energy, amino acids, and fatty acids. These findings provide novel insights into the molecular pathways of AHS-induced meat quality deterioration and reveal potential intervention timing for the poultry industry.

151

News (Medical) associated with Digoxin

16 May 2025

·www.prnewswire.com

Neurocrine Biosciences Presents Data Adding to the Growing Body of Evidence Demonstrating Functional and Quality of Life Improvements in Patients with Tardive Dyskinesia

Patients Treated with INGREZZA® (valbenazine) Capsules Reported Continued Improvements in Functional and Health-Related Quality of Life Measures Findings Presented at 2025 International Society for Pharmacoeconomics and Outcomes Research Conference SAN DIEGO, May 16, 2025 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the presentation of new analyses from a Phase 4 randomized withdrawal study (NCT03891862) showing patients with tardive dyskinesia who received continued treatment with INGREZZA® (valbenazine) capsules reported improvements across functional and health-related quality of life measures. These findings complement recently announced patient-reported outcome data from the Phase 4 KINECT-PRO™ study of INGREZZA, which was the first of its kind to specifically measure and report clinically meaningful improvements in the impact of tardive dyskinesia. The analyses were presented at the 2025 International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Conference in Montreal, Canada. "Tardive dyskinesia can affect patients' daily lives, causing pain and anxiety and hindering the ability to perform self-care tasks and to participate in work, family or social life," said Eiry W. Roberts, M.D., Chief Medical Officer, Neurocrine Biosciences. "These unique analyses add to the expansive body of evidence demonstrating that INGREZZA treatment can significantly improve tardive dyskinesia patients' quality of life, addressing outcomes important to both patients and healthcare providers." The analyses were conducted using data from 127 patients who participated in a Phase 4, double-blind, placebo-controlled, randomized withdrawal study. Patients received up to 80 mg of INGREZZA for eight weeks, after which they were randomized to either continue INGREZZA (n=59) or receive placebo (n=59) for an additional eight weeks. Health-related quality of life (HRQoL) was measured using the EuroQol 5-Dimension 5-Level (EQ-5D-5L), which includes five dimensions of health status (mobility, self-care, usual activities, pain/discomfort and anxiety/depression). A utility index, ranging from -0.573 to 1.0, and visual analog scale (EQ-VAS), ranging from 0 to 100, were also reported with higher scores indicating better health status. Functional impairment was measured using the five-item Sheehan Disability Scale (SDS), in which three items assessed work/school (patient had to be working or attending school to be included), social life and family/home life impairment. Scores were combined for an SDS total score for patients with work/school scores. Reductions in scores indicated improvement. In the open-label portion of the study, patients receiving INGREZZA treatment for eight weeks experienced significant improvements from baseline in multiple areas of HRQoL (Poster #PCR191), including mobility (change from baseline: -0.27), self-care (-0.28), usual activities (-0.36) and pain/discomfort (-0.34). Those randomized to receive INGREZZA for an additional eight weeks (Week 16) saw continued improvements in all HRQoL dimensions, including significant improvements in mobility (placebo-adjusted difference from Week 8: -0.34) and anxiety/depression (-0.38) compared with those receiving placebo. In a separate analysis (Poster #PCR190), patients in the open-label portion of the same study receiving INGREZZA treatment for eight weeks experienced significant improvements from baseline in work/school (change from baseline: -1.37), social life (-1.65), family/home life (-1.30) and SDS total score (-4.28). Those randomized to receive INGREZZA for an additional eight weeks saw continued improvements in all domains, including significant improvements in social life (placebo-adjusted difference from Week 8: -0.95) and family/home life (-0.89) compared with those receiving placebo. Additional presentations at the 2025 ISPOR annual meeting include: Assessing Real-World Dosing Patterns for Vesicular Monoamine Transporter-2 Inhibitors, Valbenazine and Deutetrabenazine, Among Patients with Tardive Dyskinesia in a Nationwide US Claims Database (Poster #HSD80) Disparities in Tardive Dyskinesia Diagnosis and Treatment Among the US Medicare Population (Poster #HSD59) Disparities in Antipsychotic Prescribing Among the US Medicare Population (Podium Presentation) The Impact of Glucocorticoid Dose on Clinical Outcomes in Congenital Adrenal Hyperplasia: a Systematic Literature Review (Poster #CO16) Indirect Treatment Comparison for Early Efficacy of VMAT2 Inhibitors for Tardive Dyskinesia and Chorea Associated with Huntington's Disease (Poster #CO113) About Tardive Dyskinesia Tardive dyskinesia (TD) is a movement disorder that is characterized by uncontrolled, abnormal and repetitive movements of the face, torso and/or other body parts, which may be disruptive and negatively impact patients. The condition is associated with taking certain kinds of mental health medicines (antipsychotics) that help control dopamine receptors in the brain. Taking antipsychotics commonly prescribed to treat mental illnesses such as major depressive disorder, bipolar disorder, schizophrenia and schizoaffective disorder and other prescription medicines (metoclopramide and prochlorperazine) used to treat gastrointestinal disorders are associated with TD. In patients with TD, these treatments are thought to result in irregular dopamine signaling in a region of the brain that controls movement. The symptoms of TD can be mild to severe and are often persistent and irreversible. TD is estimated to affect at least 800,000 adults in the U.S. About Chorea Associated with Huntington's Disease Huntington's disease (HD) is a hereditary progressive neurodegenerative disorder in which the loss of certain neurons within the brain causes motor, cognitive and psychiatric symptoms. Symptoms generally appear between the ages of 30 and 50 years and worsen over a 10- to 25-year period. Most people with HD experience chorea, an abnormal involuntary movement disorder, characterized by irregular and unpredictable movements. Chorea can affect various body parts and interfere with motor coordination, gait, swallowing and speech. HD is estimated to affect approximately 41,000 adults in the U.S., with more than 200,000 at risk of inheriting the disease. About INGREZZA®  (valbenazine) Capsules and INGREZZA®  SPRINKLE (valbenazine) Capsules INGREZZA is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor approved by the U.S. Food and Drug Administration for the treatment of adults with tardive dyskinesia and the treatment of chorea associated with Huntington's disease (HD). Only INGREZZA offers a therapeutic dose from day one with no required titration. INGREZZA, developed by Neurocrine Biosciences, selectively inhibits VMAT2 with no appreciable binding affinity for VMAT1, dopaminergic (including D2), serotonergic, adrenergic, histaminergic or muscarinic receptors. While the specific way INGREZZA works to treat TD and HD chorea is not fully understood, INGREZZA is unique in that it selectively and specifically targets VMAT2 to inhibit the release of dopamine, a chemical in the brain that helps control movement. INGREZZA is believed to reduce extra dopamine signaling, which may lead to fewer uncontrollable movements. INGREZZA is proven across the widest range of patients. It is always one capsule, once daily and can be taken together with most stable mental health regimens such as antipsychotics or antidepressants. Only INGREZZA offers the benefit of a sprinkle formulation, INGREZZA SPRINKLE, for those who experience dysphagia, have difficulty swallowing or prefer not to swallow a pill. INGREZZA and INGREZZA SPRINKLE dosages approved for use are 40 mg, 60 mg and 80 mg capsules. Important Information Approved Uses INGREZZA® (valbenazine) capsules or INGREZZA® SPRINKLE (valbenazine) capsules are prescription medicines used to treat adults with: movements in the face, tongue, or other body parts that cannot be controlled (tardive dyskinesia). involuntary movements (chorea) of Huntington's disease. INGREZZA or INGREZZA SPRINKLE do not cure the cause of involuntary movements, and do not treat other symptoms of Huntington's disease, such as problems with thinking or emotions. It is not known if INGREZZA or INGREZZA SPRINKLE is safe and effective in children. IMPORTANT SAFETY INFORMATION INGREZZA or INGREZZA SPRINKLE can cause serious side effects in people with Huntington's disease, including: depression, suicidal thoughts, or suicidal actions. Tell your healthcare provider before you start taking INGREZZA or INGREZZA SPRINKLE if you have Huntington's disease and are depressed (have untreated depression or depression that is not well controlled by medicine) or have suicidal thoughts. Pay close attention to any changes, especially sudden changes, in mood, behaviors, thoughts, or feelings. This is especially important when INGREZZA or INGREZZA SPRINKLE is started and when the dose is changed. Call your healthcare provider right away if you become depressed, have unusual changes in mood or behavior, or have thoughts of hurting yourself. Do not take INGREZZA or INGREZZA SPRINKLE if you: are allergic to valbenazine, or any of the ingredients in INGREZZA or INGREZZA SPRINKLE. INGREZZA or INGREZZA SPRINKLE can cause serious side effects, including: Allergic reactions. Allergic reactions, including an allergic reaction that causes sudden swelling called angioedema, can happen after taking the first dose or after many doses of INGREZZA or INGREZZA SPRINKLE. Signs and symptoms of allergic reactions and angioedema include: trouble breathing or shortness of breath, swelling of your face, lips, eyelids, tongue, or throat, or other areas of your skin, trouble with swallowing, or rash, including raised, itchy red areas on your skin (hives). Swelling in the throat can be life-threatening and can lead to death. Stop taking INGREZZA or INGREZZA SPRINKLE and go to the nearest emergency room right away if you develop these signs and symptoms of allergic reactions and angioedema. Sleepiness and tiredness that could cause slow reaction times (somnolence and sedation). Do not drive a car or operate dangerous machinery until you know how INGREZZA or INGREZZA SPRINKLE affects you. Drinking alcohol and taking other medicines may also cause sleepiness during treatment with INGREZZA or INGREZZA SPRINKLE. Heart rhythm problems (QT prolongation). INGREZZA or INGREZZA SPRINKLE may cause a heart rhythm problem known as QT prolongation. You have a higher chance of getting QT prolongation if you also take certain other medicines during treatment with INGREZZA or INGREZZA SPRINKLE. Tell your healthcare provider right away if you develop any signs or symptoms of QT prolongation, including: fast, slow, or irregular heartbeat (heart palpitations), shortness of breath, dizziness or lightheadedness, or fainting or feeling like you are going to faint. Neuroleptic Malignant Syndrome (NMS). NMS is a serious condition that can lead to death. Call a healthcare provider right away or go to the nearest emergency room if you develop these symptoms and they do not have another obvious cause: high fever, stiff muscles, problems thinking, irregular pulse or blood pressure, increased sweating, or very fast or uneven heartbeat. Parkinson-like symptoms. Symptoms include: body stiffness, drooling, trouble moving or walking, trouble keeping your balance, shaking (tremors), or falls. Before taking INGREZZA or INGREZZA SPRINKLE, tell your healthcare provider about all of your medical conditions including if you: have liver or heart problems, are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Make sure you tell all of your healthcare providers that you are taking INGREZZA or INGREZZA SPRINKLE. Taking INGREZZA or INGREZZA SPRINKLE with certain other medicines may cause serious side effects. Especially tell your healthcare provider if you: take digoxin or take or have taken a monoamine oxidase inhibitor (MAOI) medicine. You should not take INGREZZA or INGREZZA SPRINKLE if you are taking, or have stopped taking, a MAOI within the last 14 days. The most common side effects of INGREZZA or INGREZZA SPRINKLE in people with tardive dyskinesia  are sleepiness and tiredness. The most common side effects of INGREZZA or INGREZZA SPRINKLE in people with chorea associated with Huntington's disease include sleepiness and tiredness, raised itchy red areas on your skin (hives), rash, and trouble getting to sleep or staying asleep. These are not all of the possible side effects of INGREZZA or INGREZZA SPRINKLE. Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch at   or call  1-800-FDA-1088. Dosage Forms and Strengths: INGREZZA and INGREZZA SPRINKLE are available in 40 mg, 60 mg, and 80 mg capsules. Please see full Prescribing Information, including Boxed Warning, and Medication Guide . About Neurocrine Biosciences, Inc. Neurocrine Biosciences is a leading neuroscience-focused, biopharmaceutical company with a simple purpose: to relieve suffering for people with great needs. We are dedicated to discovering and developing life-changing treatments for patients with under-addressed neurological, neuroendocrine and neuropsychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington's disease, classic congenital adrenal hyperplasia, endometriosis* and uterine fibroids,* as well as a robust pipeline including multiple compounds in mid- to late-phase clinical development across our core therapeutic areas. For three decades, we have applied our unique insight into neuroscience and the interconnections between brain and body systems to treat complex conditions. We relentlessly pursue medicines to ease the burden of debilitating diseases and disorders because you deserve brave science. For more information, visit neurocrine.com, and follow the company on LinkedIn, X and Facebook. (*in collaboration with AbbVie) The NEUROCRINE BIOSCIENCES Logo, NEUROCRINE, YOU DESERVE BRAVE SCIENCE and INGREZZA are registered trademarks of Neurocrine Biosciences, Inc. KINECT-PRO is a trademark of Neurocrine Biosciences, Inc. Forward-Looking Statements In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. These statements include, but are not limited to, statements regarding the potential benefits to be derived from INGREZZA and the value INGREZZA may bring to patients. Factors that could cause actual results to differ materially from those stated or implied in the forward-looking statements include, but are not limited to, the following: risks and uncertainties associated with Neurocrine Biosciences' business and finances in general, as well as risks and uncertainties associated with the commercialization of INGREZZA; whether INGREZZA receives adequate reimbursement from third-party payors; risks and uncertainties relating to competitive products and technological changes that may limit demand for INGREZZA; risks associated with the Company's dependence on third parties for development and manufacturing activities related to INGREZZA, and the ability of the Company to manage these third parties; risks that additional regulatory submissions for INGREZZA or other product candidates may not occur or be submitted in a timely manner; risks that the FDA or other regulatory authorities may make adverse decisions regarding INGREZZA; risks that post-approval INGREZZA commitments or requirements may be delayed; risks that INGREZZA may be precluded from commercialization by the proprietary or regulatory rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; and other risks described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's quarterly report on Form 10-Q for the quarter ended March 31, 2025. Neurocrine Biosciences disclaims any obligation to update the statements contained in this press release after the date hereof other than required by law. © 2025 Neurocrine Biosciences, Inc. All Rights Reserved. CAP-VBZ-US-0064 05/2025 SOURCE Neurocrine Biosciences, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultPhase 3Drug Approval

16 May 2025

·www.globenewswire.com

BioCryst Presents New Real-world Evidence Showing Significant and Sustained Reductions in HAE Attack Rates in Adolescents and People with Severe HAE Following Initiation of ORLADEYO® (berotralstat)

RESEARCH TRIANGLE PARK, N.C., May 16, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new real-world evidence on the use of oral, once-daily ORLADEYO® (berotralstat) in adolescents and people with severe HAE showing significant and sustained reductions in HAE attack rates through 18 months of follow-up after beginning treatment with ORLADEYO in both patient populations. The real-world evidence was presented in two posters at the 2025 International Society for Pharmacoeconomics and Outcomes Research conference (ISPOR), which is being held in Montreal from May 13-16, 2025. “The outcomes detailed in these posters show how ORLADEYO is making a difference for people living with HAE, in particular those with very severe disease and those who are adolescents. These two groups experienced far fewer attacks per month compared to baseline after starting ORLADEYO. These kinds of real-world results should give physicians as well as their HAE patients the additional confidence to improve control of their attacks,” said Dr. Raffi Tachdjian, associate clinical professor of medicine & pediatrics, division of allergy & clinical immunology, David Geffen School of Medicine, University of California Los Angeles. Significant and sustained reductions in attack rates after ORLADEYO initiation The results presented in two posters at ISPOR 2025 were from a retrospective pre-post study using outcomes collected from BioCryst’s sole-source pharmacy from December 15, 2020, to January 8, 2024. The poster “Real-World Hereditary Angioedema Attack Rates Before and After Berotralstat Initiation Among Patients with C1 Inhibitor Deficiency (Type I/II) and ≥8 Attacks/month” (#PCR182) detailed findings from 56 U.S. patients with HAE with C1-inhibitor deficiency (HAE-C1-INH) who received ORLADEYO. Patients experienced significantly lower HAE attack rates while on ORLADEYO in each 90-day follow-up period (1.24-1.90 attacks/month) compared to baseline (7.78-8.23 attacks/month).Patients experienced significantly fewer HAE attacks per month following ORLADEYO initiation during every 90-day follow-up period relative to baseline, including: 6.25 fewer attacks/month (95 percent confidence period (CI): [5.63, 6.87]; p<0.001) at 12 months (days 271-360)6.43 fewer attacks/month (95 percent CI: [5.78, 7.09]; p<0.001) at 18 months (days 451-540) The poster “Real-World Hereditary Angioedema Attack Rates Before and After Berotralstat Initiation Among Adolescents” (#PCR132) highlighted outcomes reported from 99 U.S. patients with HAE aged 12-17 years who received ORLADEYO. Patients had significantly lower HAE attack rates while on ORLADEYO during each 90-day follow-up period (0.36-0.76 attacks/month) compared to the mean baseline rate (2.07-2.30 attacks/month).Compared to baseline, adolescents experienced statistically significant and sustained reductions in HAE attack rates after ORLADEYO initiation during each 90-day follow-up period, including: 1.56 fewer attacks/month (95 percent CI: [0.89, 2.23]; p<0.001) at 12 months (days 271-360)1.85 fewer attacks/month (95 percent CI: [1.12, 2.58]; p<0.001) at 18 months (days 451-540) “We continue to generate evidence from real-world use of our oral, once-daily prophylactic therapy for HAE that supports its effectiveness in a wide range of people with HAE. Here, we show that ORLADEYO is having a positive impact on attack reduction for younger people and those with severe disease. These additional findings further underscore that ORLADEYO works well for many patients with HAE, regardless of their attack severity, age or other aspects,” said Dr. Donald S. Fong, chief medical officer of BioCryst. About ORLADEYO® (berotralstat)ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein. U.S. Indication and Important Safety Information INDICATIONORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. Limitations of useThe safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation. IMPORTANT SAFETY INFORMATIONAn increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent. The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease. A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C). Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. P-gp inducers (eg, rifampin, St. John’s wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO. ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO. The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been established. There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production. To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Please see full Prescribing Information. About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit www.biocryst.com or follow us on LinkedIn. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding future results, performance or achievements and statements relating to ORLADEYO performance and effectiveness. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst’s ability to successfully implement or maintain its commercialization plans for ORLADEYO; the commercial viability of ORLADEYO, including its ability to achieve sustained market acceptance; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay, or withdraw market approval for products and product candidates; and BioCryst’s ability to successfully manage its growth and compete effectively. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause the actual results to differ materially from those contained in BioCryst’s forward-looking statements. BCRXW Contact:John Bluth+1 919 859 7910jbluth@biocryst.com

Clinical ResultClinical Study

08 May 2025

·www.echemi.com

1 Indian Manufacturer Banned from Exporting Heart Drug to China Over Safety Violations

China’s drug regulator has suspended imports of digoxin API from Indian firm VITAL LABORATORIES PVT. LTD., citing serious quality control lapses discovered during a recent on-site inspection. The decision, effective April 27, stems from findings that the company’s actual production process deviated from what was officially registered, with insufficient research on key manufacturing changes. Regulators found that the active pharmaceutical ingredient (API) failed to meet China’s GMP standards, with issues including inadequate quality assurance systems, improper release testing, and unauthorized modifications to critical parameters. These violations conflict with China’s regulatory requirements for drug manufacturing and import oversight. Digoxin, a cardiac glycoside, is used to treat heart conditions such as atrial fibrillation, atrial flutter, and congestive heart failure. While effective, its safety relies heavily on precise manufacturing controls, making regulatory compliance especially critical. The National Medical Products Administration (NMPA) has ordered all customs offices to halt clearance for this ingredient and prohibited its use in finished drug production. Additionally, any finished products already released using this API must be investigated immediately by the Marketing Authorization Holders (MAHs), who are required to carry out risk assessments and implement appropriate safety measures. The digoxin API’s review status on China’s pharmaceutical registry has also been downgraded to “I,” indicating it has failed the joint evaluation process with its related drug products. This move effectively removes the API’s eligibility for use in domestic pharmaceutical manufacturing. This case underscores China’s zero-tolerance stance on drug safety and its ongoing efforts to tighten import controls on foreign APIs to protect public health.

100 Deals associated with Digoxin

External Link

KEGG	Wiki	ATC	Drug Bank
D00298	Digoxin	C01AA05	DB00390

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Arrhythmias, Cardiac	China	Shanghai Harvest Pharmaceutical Co., Ltd	01 Jan 1995
Acute congestive heart failure	Japan	Taiyo Pharma Co., Ltd.	10 Sep 1957
Tachycardia	Japan	Taiyo Pharma Co., Ltd.	10 Sep 1957
Atrial Fibrillation	United States	Covis Pharma Holdings BV	16 Nov 1954
Heart Failure	United States	Covis Pharma Holdings BV	16 Nov 1954

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
HIV Infections	Phase 1	United States	ViiV Healthcare Ltd.	16 Dec 2020
Chemotherapy-induced nausea and vomiting	Phase 1	United States	Tesaro, Inc.	01 May 2015
Epilepsy	Phase 1	United States	GSK Plc	16 Jan 2012
Diabetes Mellitus, Type 2	Phase 1	United States	GSK Plc	11 Jun 2010
Metastatic human epidermal growth factor 2 positive carcinoma of breast	Phase 1	United States	GSK Plc	23 Apr 2008
Metastatic human epidermal growth factor 2 positive carcinoma of breast	Phase 1	Canada	GSK Plc	23 Apr 2008

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03928210 (Nature) Manual	Early Phase 1	Metastatic breast cancer	9	Digoxin	nsqedivkkn(zravauekqh) = rheqnmuhpj gpmvdundxx (yazxoemwxe ) View more	Positive	24 Jan 2025
NCT05540600 (DIGOBET-AF, Pubmed \| Am J Cardiovasc Drugs)	Phase 3	Atrial Fibrillation	60	Digoxin 0.25 mg	jqopmysgjm(mggtohllry) = sqmuutypin qvqqoeevfz (xwfwechqqj, 3.2)	Positive	26 Dec 2024
				Bisoprolol 5-10 mg	jqopmysgjm(mggtohllry) = ukufckvkiz qvqqoeevfz (xwfwechqqj, 3.4)
NCT04834557 (Pubmed \| Front Pharmacol)	Phase 2	Rheumatoid Arthritis IL-17A \| IL-23 \| HIF-1α ... View more	60	Digoxin 0.25 mg every other day	doxtbjkfjz(culyqwnkzo) = vmcecuqwob xxdskhkubg (rfypvworpp )	Positive	21 Oct 2024
				Placebo	doxtbjkfjz(culyqwnkzo) = gvoludgkpu xxdskhkubg (rfypvworpp )
ESC2024	Not Applicable	Chronic heart failure \| Atrial Fibrillation	-	Digoxin	kztgtyfvyd(uhstidpawf) = dcykllpsjm rzhyvnrtby (uqsjmzvfdq, 17.2)	-	30 Aug 2024
ESC2024	Not Applicable	Atrial Fibrillation	-	Intravenous Digoxin Loading Dose	elcerktlpf(txtlcffqjl) = bnscvuuryc tgimgnnskl (ntqkcjwzoh, 0.9 - 1.7) View more	-	30 Aug 2024
NCT03559868 (CTgov)	Phase 1	Inflammation	45	digoxin (Digoxin 3 mcg/Kg/Day)	ktetoejjxh(qesjrzpmrc) = xgwvkprurs ugqpxrmydn (rxdamxzyui, 237) View more	-	12 Jul 2024
				digoxin (Digoxin 0.15 mcg)	ktetoejjxh(qesjrzpmrc) = xcekkhoosc ugqpxrmydn (rxdamxzyui, 483) View more
ESC2023	Not Applicable	-	-	Digoxin	scpaltivqt(mpefqewpym) = mgpcyembff xlpwasbwvb (vhczkpkksq ) View more	-	21 May 2023
NCT03909529 (CTgov)	Phase 1	Medication interactions	28	Carospir+digoxin (Drug-Drug Interaction Study)	fpppeewdgc(nnzbnptzcb) = irxyrwtymm zcieqhfmvn (mxioysezlu, 502.810) View more	-	01 May 2023
				Digoxin (LANOXIN) (Treatment-A- Single Dose of Digoxin (LANOXIN))	txlnojftde(cmbjdptwtm) = empkbhremj kijnvuknrq (ncqyaqjfad, lbwjlqnyza - psjwyclhgm) View more
NCT02391337 (RATE-AF, ESC 12022 \| ESC 22022) Manual	Phase 4	Heart failure with normal ejection fraction \| Atrial Fibrillation	130	Diazepam	sxilxvmaou(oonrnasmls): AMD = 1.1 (95% CI, 1.0 - 1.2), P-Value = 0.003 View more	Positive	29 Aug 2022
				beta-blockers
NCT04425902 (CTgov)	Phase 1	HIV Infections	20	pravastatin+omeprazole+metoprolol+midazolam+montelukast+digoxin+caffeine+flurbiprofen (Treatment A: Probe Substrates)	zfuqdkgmvb(jymdedmmoo) = jmqnwswocr eaowfsvfbz (zvqymiftgn, 31.1) View more	-	23 May 2022
				GSK3640254 (Treatment C: Probe Substrates + GSK3640254 200 mg)	zfuqdkgmvb(jymdedmmoo) = btutcgtrut eaowfsvfbz (zvqymiftgn, 32.4) View more

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