Last update 21 Nov 2024

Clobazam

Last update 21 Nov 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

1-phenyl-5-methyl-8-chloro-1,2,4,5-tetrahydro-2,4-dioxo-3H-1,5-benzodiazepine, 7-Chloro-1-methyl-5-phenyl-1,5-dihydro-benzo[b][1,4]diazepine-2,4-dione, CLB

+ [18]

Target

GABAA receptor

Mechanism

GABAA receptor agonists(Gamma-aminobutyric acid A receptor agonists)

Therapeutic Areas

Nervous System DiseasesCongenital DisordersOther Diseases

Active Indication

Lennox Gastaut SyndromeSeizuresAnxiety Disorders+ [1]

Inactive Indication

Epilepsies, MyoclonicEpilepsy, Generalized

Originator Organization

Sanofi-Aventis Australia Pty Ltd.

Active Organization

Sanofi-Aventis Australia Pty Ltd.Otter Pharmaceuticals LLCLundbeck Pharmaceuticals Ltd.

+ [2]

Inactive Organization

Lundbeck LLC

H. Lundbeck A/S

Drug Highest PhaseApproved

First Approval Date

AU (01 Aug 1991),

Anxiety DisordersEpilepsy

RegulationOrphan Drug (US), Priority Review (CN)

Structure

Molecular FormulaC16H13ClN2O2

InChIKeyCXOXHMZGEKVPMT-UHFFFAOYSA-N

CAS Registry22316-47-8

Clinical Trials associated with Clobazam

ChiCTR2400088523 / SuspendedPhase 4IIT

Observing the efficacy and safety of clobazam addition in the treatment of Dravet syndrome: a randomized, double-blind, placebo-controlled study

Start Date26 Aug 2024

Sponsor / Collaborator-

CTR20240740 / RecruitingNot Applicable

氯巴占口服混悬液（10 mg/5mL和2.5 mg/mL，20mg）在健康受试者中空腹及餐后条件下的单剂量、两周期、双交叉生物等效性试验

[Translation] Single-dose, two-period, double-crossover bioequivalence study of clobazam oral suspension (10 mg/5 mL and 2.5 mg/mL, 20 mg) in healthy subjects under fasting and fed conditions

考察空腹及餐后条件下单次口服20mg由上海爱的发制药有限公司提供的氯巴占口服混悬液（受试制剂，商品名：Tapclob®，规格：10mg/5mL）或单次口服20mg由Lundbeck Pharmaceuticals LLC持证的氯巴占口服混悬液（参比制剂，商品名：Onfi®，规格：2.5mg/mL）的生物等效性。

[Translation]

The bioequivalence of a single oral dose of 20 mg of clobazam oral suspension provided by Shanghai Aifa Pharmaceutical Co., Ltd. (test preparation, trade name: Tapclob®, strength: 10 mg/5 mL) or a single oral dose of 20 mg of clobazam oral suspension certified by Lundbeck Pharmaceuticals LLC (reference preparation, trade name: Onfi®, strength: 2.5 mg/mL) was investigated under fasting and postprandial conditions.

Start Date19 Jul 2024

Sponsor / Collaborator

Shanghai Aidefa Pharmacy Co. Ltd.

ChiCTR2400084153 / SuspendedPhase 4IIT

A randomized, double-blind, placebo-controlled, multicenter clinical study of clobazam plus for the treatment of drug-resistant epilepsy in adults - A multicenter study of the addition of clobazam to the treatment of refractory epilepsy in adults

Start Date15 May 2024

Sponsor / Collaborator-

100 Clinical Results associated with Clobazam

100 Translational Medicine associated with Clobazam

100 Patents (Medical) associated with Clobazam

1,417

Literatures (Medical) associated with Clobazam

01 Dec 2024·Food Chemistry: X

A clenbuterol detection method based on magnetic separation up-conversion fluorescent probe

Article

Author: Wu, Yuan-Feng ; Ye, Fei ; Shentu, Xuping ; Song, Xin-Jie ; Zhang, Yao ; Yu, Xiaoping ; Li, Wei ; Sun, Juan

In this study, a fluorescence detection method combining aptamer-modified up-conversion nanoparticles (UCNPs) and magnetic nanoparticles (MNPs) was developed for detection of Clenbuterol (CLB). The aptamer-modified magnetic NPs captured CLB, which reacted with the aptamer-modified UCNPs and generated a sandwich complex. The aptamer-modified UCNPs acted as a fluorescence source. The MNP-CLB-UCNP complex was retrieved from the solution using an magnetic field, and the fluorescence intensity was detected by fluorescence spectrophotometry with excitation and emission spectra at 980 nm and in the 400-800 nm region, respectively. The results showed that the fluorescence intensity gradually increased with increasing concentrations of CLB with a good specify. The method was highly sensitive for the quantification of CLB, with a limit of detection of 0.304 ng mL^-1. The recovery rate of CLB from pork samples ranged from 84 % to 94.87 %. This fluorescence method enables the sensitive, precise, and accurate quantification of CLB residues in pork samples.

05 Nov 2024·Toxicology Research

Upregulation of ACSL, ND75, Vha26 and sesB genes by antiepileptic drugs resulted in genotoxicity in drosophila

Article

Author: Shamapari, R ; Nagaraj, K

AbstractClobazam (CLB) and Vigabatrin (VGB) are commonly used antiepileptic drugs (AEDs) in the treatment of epilepsy. Here, we have examined the genotoxic effect of these AEDs in Drosophila melanogaster. The Drosophila larvae were exposed to different concentrations of CLB and VGB containing food media. The assessment encompassed oxidative stress, DNA damage, protein levels, and gene expression profiles. In the CLB-treated group, a reduction in reactive oxygen species (ROS) and lipid peroxidation (LPO) levels was observed, alongside increased levels of superoxide dismutase (SOD), catalase (CAT), and nitric oxide (NO). Conversely, the VGB-treated group displayed contrasting results, with increased ROS and LPO and decreased SOD, CAT, and NO levels. However, both CLB and VGB induced DNA damage in Drosophila. Proteomic analysis (SDS-PAGE and OHRLCMS) in the CLB and VGB groups identified numerous proteins, including Acyl-CoA synthetase long-chain, NADH–ubiquinone oxidoreductase 75 kDa subunit, V-type proton ATPase subunit E, ADP/ATP carrier protein, malic enzyme, and DNA-binding protein modulo. These proteins were found to be associated with pathways like growth promotion, notch signaling, Wnt signaling, neuromuscular junction (NMJ) signaling, bone morphogenetic protein (BMP) signaling, and other GABAergic mechanisms. Furthermore, mRNA levels of ACSL, ND75, Vha26, sesB, and Men genes were upregulated in both CLB and VGB-treated groups. These findings suggest that CLB and VGB could have the potential to induce genotoxicity and post-transcriptional modifications in humans, highlighting the importance of monitoring their effects when used as AEDs.

01 Nov 2024·Brain and Behavior

Cannabidiol Treatment for Adult Patients with Drug‐Resistant Epilepsies: A Real‐World Study in a Tertiary Center

Article

Author: Pichit, Phintip ; Calonge, Quentin ; Dupont, Sophie ; Besnard, Aurore ; Gourfinkel‐An, Isabelle ; Bailly, Laurent ; Navarro, Vincent ; Damiano, Maria

ABSTRACTBackground and purposeAround 30% of patients with epilepsy show drug‐resistant epilepsy (DRE). While cannabidiol has demonstrated efficacy as an adjunctive treatment in Dravet syndrome (DS), Lennox–Gastaut Syndrome (LGS), and epilepsy related to tuberous sclerosis complex (TSC), its more global effectiveness in adult patients with DRE apart from these three specific contexts needs to be clarified.MethodsWe conducted a retrospective study at the epilepsy unit of Pitié Salpêtrière Hospital. Patients initiating pharmaceutical cannabidiol treatment and followed for at least 1 year were included. Patients were categorized into “authorized” (LGS, DS, or TSC) and “off‐label” groups. Cannabidiol effectiveness and tolerance were compared between groups, and characteristics of responders (patients with >50% reduction in seizure frequency) in the off‐label group were examined.ResultsNinety‐one patients, followed by a median duration of 24 months, were included. A total of 35.2% of the patients were in the authorized group. No significant differences were observed in responder rates between groups (31.3% vs. 35.6%, p = 0.85) and retention rates at 1 year (75.0% vs. 74.6%, p = 0.97). Sleepiness was more commonly reported in the authorized group (50.0% vs. 22.0%, p = 0.01), with no other significant differences. Among off‐label patients (n = 59), clobazam co‐prescription was more prevalent in responders (71.4% vs. 28.9%, p = 0.002).ConclusionOur findings suggest that cannabidiol may benefit all adult patients with DRE, particularly those already receiving clobazam. Randomized controlled trials are warranted in off‐label patients to validate these observational findings.

News (Medical) associated with Clobazam

14 Nov 2024

·www.prnewswire.com

New Development in Rare Pediatric Epilepsy: Biocodex Announces the Development of DIACOMIT® (stiripentol) Oral Suspension, a New Formulation to Aid in the Treatment of Dravet Syndrome in Young Children

BEDMINSTER, N.J., Nov. 14, 2024 /PRNewswire/ -- The global pharmaceutical company, Biocodex, a long-time pioneer in the treatment of rare pediatric diseases, is proud to announce they are developing a new oral-suspension formulation for DIACOMIT, an FDA-approved treatment indicated for the management of seizures associated with Dravet syndrome. This new formulation will be indicated for patients 6 months and older (weighing 15 lb or more) taking clobazam.1 Biocodex's Research and Development (R&D) center in France is developing this latest innovation, which represents a significant advancement for young patients suffering from this rare, severe, and treatment-resistant form of epilepsy. A 100% Biocodex Innovation The new DIACOMIT oral suspension formulation is the result of years of dedicated research at Biocodex's R&D center. This innovation underscores Biocodex's ongoing commitment to address the needs of patients with rare diseases, particularly in pediatric epilepsy, where Biocodex led the first clinical trial for Dravet syndrome. Through continuous research and development, Biocodex remains at the forefront of improving care for those affected by this challenging condition. "At Biocodex Orphan, our priority is to improve the quality of life for children affected by Dravet syndrome and their families at every stage of comprehensive care. This new development is a concrete testament to our commitment to providing innovative solutions tailored to children suffering from this syndrome," said Caroline Schreiner, Vice President of Global Orphan Diseases at Biocodex. Since the inception of Biocodex Orphan, the company has reaffirmed its commitment to expanding its portfolio of treatments for rare pediatric diseases, particularly in neuropediatrics, through partnerships and innovative solutions. Meeting the Needs of Patients and Caregivers DIACOMIT is an approved Dravet specific treatment, available in over 55 countries. Currently offered in capsules and sachets formats, DIACOMIT will be available as an oral suspension, a formulation better suited to the needs of younger patients, especially infants with low body weight, those who cannot swallow capsules, or require precise dose adjustments. This new formulation will enhance administration ease, better meeting the needs of patients, caregivers, and the Dravet community. Putting Patients First Biocodex's mission extends beyond treatment solutions. The company is committed to raising awareness about rare epilepsies alongside their ecosystem partners (healthcare professionals, patients, and caregivers), providing innovative solutions and services for holistic care for the Dravet community and establishing itself as a trusted partner in their journey. About Dravet Syndrome Dravet syndrome is a rare and severe genetic epilepsy that most commonly begins before the age of one when an otherwise normally developing child begins having frequent, prolonged seizures.2 These seizures can cause lasting damage, which over time can lead to developmental and cognitive delays, affecting coordination, language, and behavior.3 Its incidence is estimated to be 1 in 16,000 births.4 About Biocodex Founded in 1953, Biocodex is a French family-owned pharmaceutical company that leverages life sciences, placing health and its balance at the heart of its corporate initiatives, innovations, and development for the benefit of patients worldwide. A pioneer and leader in human microbiota health by marketing the first probiotic strain, the global organization has developed its activities around three strategic areas: microbiota, women's health, and orphan diseases. It has also expanded its expertise by investing in other health areas, such as pain management, respiratory and ENT pathways, and neurology. Present in 115 countries through its subsidiaries and partners, Biocodex emphasizes its territorial roots, which are central to its corporate social responsibility, producing in France for over 50 years across most of its value chain. The Biocodex global organization is comprised of 1,700 employees who share common values, embodied in their daily missions and rooted in the company's DNA. To learn more about Biocodex visit: To learn more about DIACOMIT visit: INDICATION DIACOMIT (stiripentol) is indicated for the treatment of seizures associated with Dravet syndrome (DS) in patients taking clobazam who are 6 months of age and older and weighing 7 kg or more. There are no clinical data to support the use of DIACOMIT as monotherapy in Dravet syndrome. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS None WARNINGS & PRECAUTIONS Somnolence DIACOMIT can cause somnolence. Monitor patients for somnolence, particularly when DIACOMIT is used concomitantly with other CNS depressants or clobazam, which is also known to cause somnolence. Decreased Appetite and Decreased Weight DIACOMIT can cause decreases in appetite and weight. The growth and weight of pediatric patients treated with DIACOMIT should be carefully monitored. Neutropenia and Thrombocytopenia DIACOMIT can cause significant declines in neutrophil and platelet counts. Hematologic testing should be obtained prior to starting treatment with DIACOMIT and then every 6 months. Withdrawal Symptoms As with most antiepileptic drugs (AEDs), DIACOMIT should be gradually withdrawn to minimize the risk of increased seizure frequency and status epilepticus. Risks in Patients with Phenylketonuria (PKU) DIACOMIT for oral suspension contains phenylalanine, which can be harmful to patients with PKU. Before prescribing DIACOMIT for oral suspension to a patient with PKU, consider the total daily intake of phenylalanine from all sources, including DIACOMIT for oral suspension. DIACOMIT capsules do not contain phenylalanine. Suicidal Behavior and Ideation AEDs, including DIACOMIT, increase the risk of suicidal thoughts or behavior. Patients treated with any AED for any indication should be monitored for the emergence or worsening of depression, suicidal thoughts or behavior, and/or any unusual changes in mood or behavior. ADVERSE REACTIONS The most common adverse reactions that occurred in at least 10% of DIACOMIT-treated patients and more frequently than on placebo were somnolence, decreased appetite, agitation, ataxia, decreased weight, hypotonia, nausea, tremor, dysarthria, and insomnia. PREGNANCY There are no adequate data on the developmental risks associated with the use of DIACOMIT in pregnant women. Based on animal data, DIACOMIT may cause fetal harm. There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to AEDs, such as DIACOMIT, during pregnancy. Physicians are advised to recommend that pregnant patients taking DIACOMIT enroll in the North American Antiepileptic Drug (NAAED) Pregnancy Registry (information at ). This can be done by calling the toll-free number 1‑888‑233-2334 and must be done by patients themselves or their caregiver. To report suspected adverse reactions, contact Biocodex at 1‑866‑330-3050 or FDA at 1‑800‑FDA‑1088 or . Please see full DIACOMIT Prescribing Information at . References: 1. DIACOMIT® [prescribing information]. Beauvais, France: Biocodex, Inc.; July 2022 2. Dravet C. The core Dravet syndrome phenotype. Epilepsia. 2011;52 Suppl 2:3-9. doi:10.1111/j.1528-1167.2011.02994. 3. Genton P, Velizarova R, Dravet C. Dravet syndrome: the long-term outcome. Epilepsia. 2011;52 Suppl 2:44-49. doi:10.1111/j.1528-1167.2011.03001. 4. Wu YW, Sullivan J, McDaniel SS, et al. Incidence of Dravet Syndrome in a US Population. Pediatrics. 2015;136(5):e1310-e1315. doi:10.1542/peds.2015-1807 © 2024 Biocodex, Inc. All rights reserved. DIACOMIT® is a registered trademark of Biocodex, Inc. SOURCE Biocodex, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug ApprovalOrphan DrugClinical Study

05 Sep 2024

·www.prnewswire.com

Jazz Pharmaceuticals to Present New Data Demonstrating Improved Epilepsy Outcomes with Epidiolex®/Epidyolex® (cannabidiol) at the European Epilepsy Congress 2024

Data from eight abstracts to be presented, including results of the BECOME (BEhavior, COgnition and More with Epidyolex®) Caregiver Survey on the real-world outcomes of Epidyolex® (cannabidiol) treatment on patients with Lennox-Gastaut Syndrome (LGS) and Dravet Syndrome (DS) DUBLIN, Sept. 5, 2024 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the Company will present eight abstracts at the 15th European Epilepsy Congress (EEC), including a subgroup analysis of the BECOME (BEhavior, COgnition and More with Epidyolex®) Caregiver Survey, a survey of caregivers of patients with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) reporting outcomes in patients receiving Epidiolex/Epidyolex (cannabidiol, 100mg/mL oral solution) and concomitant clobazam. Results showed most caregivers reported patient improvements in seizure and non-seizure outcomes since initiating Epidyolex. In addition, post-hoc analyses of pooled data from 215 participants from the GWPCARE3 and GWPCARE4 Epidyolex Phase 3 trials will be presented, demonstrating thresholds for clinically meaningful reductions in drop seizures in patients with LGS. EEC is being held September 7-11, 2024, in Rome, Italy. "Presentations at EEC this year continue to build the scientific evidence in support of Epidyolex, with data from more than 2,000 patient experiences, demonstrating improved understanding and real-world outcomes reflecting the treatment's holistic impact -- not only for patients but importantly their families and caregivers as well," said Tomas Skacel, vice president of medical affairs, Europe and international, at Jazz Pharmaceuticals. "In addition to seizure outcomes, the BECOME subgroup analysis underscores the impact of non-seizure benefits of Epidyolex and the potential to improve the day-to-day lives for both patients and their loved ones." Presentation highlights include: Epidyolex Beyond-Seizure Benefits (P251): Results from the BECOME subgroup analysis indicating that treatment with Epidyolex (for ≥3 months) and concomitant clobazam, in both pediatric (2–17 years) and adult (≥18 years) patients with LGS or DS, led to caregiver-reported improvements in both seizure frequency (87%), seizure severity (81%) and weekly seizure-free days (68%). Notably, results reported improvements in at least one item for non-seizure-related domains, including alertness, cognition and executive function (84%) and language and communication in non-verbal (81%) and verbal (76%) patients. In addition, 94% of the 243 caregivers surveyed indicated they planned to continue with Epidyolex treatment. Epidyolex Efficacy in Drop Seizures (P424): Results from a post-hoc analysis of data from pivotal trials GWPCARE3 and GWPCARE4, in patients with LGS who received Epidyolex for 14 weeks, evaluated the threshold for a clinically meaningful reduction in drop seizures associated with Caregiver Global Impression of Change (CGIC). The analysis showed that of 215 patients analyzed, 67 (31%) reported a 'much improved or better' CGIC score and 129 (60%) reported a 'slightly improved or better' CGIC score; and that the best threshold for a clinically important response (CIR) were reductions in drop seizures of −30.6% and −49.6% for 'slightly improved or better' and 'much improved or better', respectively. For those receiving concomitant clobazam, CIR thresholds were relatively consistent between subgroups and the overall population. Need for Improved Diagnosis (P293 & P292): Results from two analyses of real-world data from the Adelphi LGS and DS Disease-Specific Programmes™, which are independent real-world evidence databases. The analyses describe the clinical characteristics of 276 patients with LGS and 212 patients with DS from across Europe (France, Germany, Italy, Spain and the UK). These analyses, which were not limited to Epidyolex, suggest: Initial misdiagnosis is common in both patients with LGS and DS (53.7% of pediatric and 81.3% of adult patients were diagnosed with another seizure type prior to LGS; and 66.7% of pediatric and 79.3% of adult patients were diagnosed with another seizure type prior to DS) – highlighting the need for improved diagnosis. Following initial monotherapy, the majority of patients go on to receive at least two further anti-seizure medications (83.2% of pediatric and 82.7% of adult LGS patients subsequently received ≥2 medications; and 83.9% of pediatric and 71.4% of adult DS patients subsequently received ≥2 medications) – highlighting the need to identify effective early line treatment options. Effect of a Ketogenic Diet: A late-breaking abstract featuring the results of a post-hoc analysis of data from three randomized, controlled trials: two in LGS patients (GWPCARE3/NCT02224560 and GWPCARE4/NCT02224690), and one in DS patients (GWPCARE2/NCT02224703), who received Epidyolex for 14 weeks, which explored the effect of a ketogenic diet (KD)*, on cannabidiol plasma concentration and seizure reduction in patients with LGS or DS treated with Epidyolex in conjunction with clobazam. The analysis found no substantial differences in cannabidiol plasma concentration or seizure responses between patients in KD and non-KD groups. Further studies are needed to confirm these findings. * KD; A form of non-pharmacological treatment used in attempts to provide seizure control for patients who experience pharmacoresistance. The EEC 2024 abstracts are available at: . All Jazz Pharmaceuticals data will be featured at the in-person poster presentations, occurring Sunday, 8 September through Tuesday, 10 September, with investigators available 13:30-15:00 daily. A full list of the presentations are: About Epidiolex®/Epidyolex® (cannabidiol) Epidiolex®/Epidyolex® (cannabidiol; 100 mg/mL oral solution), a prescription, plant-derived cannabis-based medicine approved by the U.S. Food and Drug Administration (FDA) for use in the U.S, and the European Medicines Agency (EMA) for use in the European Union, is an oral solution which contains highly purified cannabidiol (CBD). In the U.S., cannabidiol is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome or Tuberous Sclerosis Complex (TSC) in patients one year of age and older. Cannabidiol has also received approval in the European Union, under the tradename Epidyolex, for adjunctive use in conjunction with clobazam to treat seizures associated with LGS and Dravet syndrome in patients two years and older, and for adjunctive use to treat seizures associated with TSC, in patients two years of age and older. About Jazz Pharmaceuticals Jazz Pharmaceuticals plc is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing potentially life-changing medicines for people with serious diseases — often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Contacts: Media: Kristin Bhavnani Head of Global Corporate Communications Jazz Pharmaceuticals plc [email protected] Ireland +353 1 637 2141 U.S. +1 215 867 4948 Investors: Andrea N. Flynn, Ph.D. Vice President, Head, Investor Relations Jazz Pharmaceuticals plc [email protected] Ireland +353 1 634 3211 U.S. +1 650 496 2717 SOURCE Jazz Pharmaceuticals plc

Clinical ResultDrug ApprovalPhase 3

22 Aug 2024

·www.prnewswire.com

Jazz Pharmaceuticals Provides Update on Cannabidiol Oral Solution Phase 3 Trial in Japan in Treatment-Resistant Epilepsies

DUBLIN, Aug. 22, 2024 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced top-line results from the Phase 3 open-label, single-arm trial in Japan evaluating the safety and efficacy of cannabidiol oral solution (marketed as Epidiolex®/Epidyolex® globally) as an adjunctive treatment for seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS) or tuberous sclerosis complex (TSC). The trial did not meet the primary efficacy endpoint of a pre-specified percentage change in indication-associated seizure frequency during the treatment period (up to 16 weeks) compared to baseline in Japanese pediatric patients; however, numeric improvements were observed in the primary and several secondary endpoints. No new safety signals were observed in the trial. "We are confident in the overall clinical profile of Epidyolex, which has been established in five Phase 3 clinical trials in more than 900 patients. We believe the totality of the Epidyolex global data, including the findings from this trial, supports advancement of the program in Japan," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development of Jazz Pharmaceuticals. "We are continuing to collect data in Japanese patients and plan to engage with regulatory authorities in Japan regarding a potential new drug application (JNDA). We recognize the significant unmet need for patients in Japan living with rare epilepsies and thank the investigators, patients and caregivers who are involved in this trial." About the Phase 3 Trial The Phase 3 open-label, single-arm clinical trial investigates the safety and efficacy of cannabidiol oral solution (GWP42003-P) for the treatment of seizures associated with LGS, DS or TSC in Japanese pediatric patients. The trial includes a pre-specified primary efficacy outcome measuring the percentage change in indication-associated seizure frequency during the treatment period (up to 16 weeks) compared to baseline in 62 patients ≥ 1 to ≤ 18 years of age. The trial design includes a treatment period consisting of an initial 2-week titration period followed by a 14-week maintenance period; and a period evaluating safety for up to 52 weeks of treatment. The trial remains ongoing to collect efficacy and safety data in pediatric and adult Japanese patients. About Cannabidiol Cannabidiol, 100 mg/mL oral solution, a prescription, plant-derived cannabis-based medicine approved by the U.S. Food and Drug Administration (FDA) for use in the U.S., and the European Medicines Agency (EMA) for use in the European Union, is an oral solution which contains highly purified cannabidiol (CBD). In the U.S., cannabidiol, under the tradename Epidiolex, is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome or tuberous sclerosis complex (TSC) in patients one year of age and older. Cannabidiol has also received approval in the European Union, under the tradename Epidyolex, for adjunctive use in conjunction with clobazam to treat seizures associated with LGS and Dravet syndrome in patients two years and older, and for adjunctive use to treat seizures associated with TSC, in patients two years of age and older. About Jazz Pharmaceuticals Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharmaceutical company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing life-changing medicines for people with serious diseases — often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit for more information. Caution Concerning Forward-Looking Statements This press release contains forward-looking statements, including, but not limited to, statements related to potential timing of data availability and regulatory engagement related to the Phase 3 clinical trial in Japan and other statements that are not historical facts. These forward-looking statements are based on Jazz Pharmaceuticals' current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with pharmaceutical product development, and other risks and uncertainties affecting Jazz Pharmaceuticals and its development programs, including those described from time to time under the caption "Risk Factors" and elsewhere in Jazz Pharmaceuticals plc's Securities and Exchange Commission filings and reports (Commission File No. 001-33500), including Jazz Pharmaceuticals' Annual Report on Form 10-K for the year ended December 31, 2023, as supplemented by our Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, and future filings and reports by Jazz Pharmaceuticals. Other risks and uncertainties of which Jazz Pharmaceuticals is not currently aware may also affect Jazz Pharmaceuticals' forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof or as of the dates indicated in the forward-looking statements, even if they are subsequently made available by Jazz Pharmaceuticals on its website or otherwise. Jazz Pharmaceuticals undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made. Contacts: Media: Kristin Bhavnani Head of Global Corporate Communications Jazz Pharmaceuticals plc [email protected] Ireland +353 1 637 2141 U.S. +1 215 867 4948 Investors: Andrea N. Flynn, Ph.D. Vice President, Head, Investor Relations Jazz Pharmaceuticals plc [email protected] Ireland +353 1 634 3211 U.S. +1 650 496 2717 SOURCE Jazz Pharmaceuticals plc

Phase 3Clinical ResultDrug Approval

100 Deals associated with Clobazam

External Link

KEGG	Wiki	ATC	Drug Bank
D01253	Clobazam	N05BA09	DB00349

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Lennox Gastaut Syndrome	US	Lundbeck Pharmaceuticals Ltd.	21 Oct 2011
Seizures	JP	Sumitomo Pharma Co., Ltd.	10 Mar 2000
Anxiety Disorders	AU	Sanofi-Aventis Australia Pty Ltd.	01 Aug 1991
Epilepsy	AU	Sanofi-Aventis Australia Pty Ltd.	01 Aug 1991

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Epilepsy, Generalized	Phase 1	BY	Lundbeck LLC	01 Aug 2007
Epilepsy, Generalized	Phase 1	LT	Lundbeck LLC	01 Aug 2007
Epilepsy, Generalized	Phase 1	US	Lundbeck LLC	01 Aug 2007
Epilepsy, Generalized	Phase 1	IN	Lundbeck LLC	01 Aug 2007
Epilepsy, Generalized	Phase 1	AU	Lundbeck LLC	01 Aug 2007
Seizures	Phase 1	IN	Lundbeck LLC	01 Aug 2007
Seizures	Phase 1	AU	Lundbeck LLC	01 Aug 2007
Seizures	Phase 1	BY	Lundbeck LLC	01 Aug 2007
Seizures	Phase 1	LT	Lundbeck LLC	01 Aug 2007
Lennox Gastaut Syndrome	Phase 1	-	Lundbeck LLC	01 Dec 2005

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


CNS2023	Not Applicable	Status Epilepticus Second line	-	Acetazolamide (AZM)	qypqtfmuml(fhdccinvcw) = unxwmtgxql gdovjctsng (lvmzknwzxy )	-	01 Oct 2023
				Benzodiazepine - diazepam or clobazam	qypqtfmuml(fhdccinvcw) = bvomvdutyd gdovjctsng (lvmzknwzxy )
IEC2023	Not Applicable	Epilepsies, Myoclonic	-	Lorazepam	ofvlyhqmcj(goopylxaac) = zpjnnmunyt xpgfjfughw (zpdjwoakzw )	-	04 Sep 2023
				Valproic acid	ofvlyhqmcj(goopylxaac) = wonbjlwyme xpgfjfughw (zpdjwoakzw )
HEP2 (HEP2, IEC2023)	Not Applicable	-	146	Levetiracetam	tocgqnclwr(uyuhavqbbb) = qjdcaxippq pjurlgjndc (jknesvkuil, 38.5%) View more	-	04 Sep 2023
				Lamotrigine	tocgqnclwr(uyuhavqbbb) = ngbjirzmyj pjurlgjndc (jknesvkuil, 43.5%) View more
AAN2021	Phase 3	Seizures	1,347	Cenobamate	tabafeqbrx(yfznrlqbll) = Clobazam doses were decreased most often due to AEs (eg, tiredness/fatigue, sleepiness/drowsiness, slurred speech) cgmbbdlebv (qvrwkatzwd )	-	13 Apr 2021
				Clobazam
NCT03371836 (CTgov)	Phase 4	Epilepsy \| Anxiety Disorders	20	Clobazam	znajflyyru(alxxjfgfwf) = xflxiypive vnrkqrgzeh (rjwxnrugim, whkqsxxole - sqbndbgvsp)	-	04 Mar 2021
2687 (AAN2020)	Not Applicable	Brain Cancer Add-on	32	Clobazam	tivgvyspmk(iuelhhwqap) = crpjvfrhyk hreprdwnmy (dsnkcfxrac )	Positive	14 Apr 2020
392 (AAN2020)	Not Applicable	Epilepsy Maintenance	210	Levetiracetam	(panzjmqppz) = 13/210 (6.2%) tpmzgxhrih (hpfuvwnsjj ) View more	Positive	14 Apr 2020
				Lamotrigine
P1.5-035 (AAN2019)	Not Applicable	Drug Resistant Epilepsy	417	Clobazam	jdmepjulea(uelvafqcqv) = talskkoiqj ffmxteuwwx (brkvgcqfue ) View more	Positive	09 Apr 2019
P1.5-031 (AAN2019)	Not Applicable	Epilepsy	196	Clobazam	nptncogqco(zscfkuwqcj) = bdmfwfpqot emrgfxidaz (pzldwfihuf ) View more	Positive	09 Apr 2019
NCT02564952 (CTgov)	Phase 2	Epilepsy	18	GWP42003-P+Clobazam	efwdytxytj(zzefwfjkth) = risfsithwo yovzoxqjjc (pvuyxfausa, bkwziuzudr - pqpwewwdgo) View more	-	11 Sep 2018

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