FDA Extends PDUFA Date of Biohaven's Troriluzole NDA for Rare Disease Spinocerebellar Ataxia
The FDA has extended the PDUFA date for Biohaven's (NYSE: BHVN) troriluzole new drug application (NDA) for treating spinocerebellar ataxia (SCA) by three months to Q4 2025. The extension allows for a full review of recent submissions, and the FDA plans to hold an advisory committee meeting. No new concerns were raised.
Troriluzole, which has received Fast-Track, Orphan Drug Designation, and Priority Review from the FDA, could become the first FDA-approved treatment for SCA, a rare genetic neurodegenerative disease. Clinical data shows troriluzole, a once-daily oral pill, slows disease progression by 50-70% and reduces fall risk. The mid-cycle review revealed no major safety concerns, and a Risk Evaluation and Mitigation Strategy (REMS) appears unnecessary.
La FDA ha prorogato la data PDUFA per la domanda di nuovo farmaco (NDA) di Biohaven (NYSE: BHVN) relativa al troriluzolo per il trattamento dell'ataxia spinocerebellare (SCA) di tre mesi, spostandola al quarto trimestre del 2025. Questa estensione consente una revisione completa delle recenti sottoposizioni, e la FDA prevede di tenere una riunione del comitato consultivo. Non sono emerse nuove preoccupazioni.
Il troriluzolo, che ha ottenuto la Designazione Fast-Track, Orphan Drug e Revisione Prioritaria dalla FDA, potrebbe diventare il primo trattamento approvato dalla FDA per la SCA, una rara malattia neurodegenerativa genetica. I dati clinici mostrano che il troriluzolo, una compressa orale da assumere una volta al giorno, rallenta la progressione della malattia del 50-70% e riduce il rischio di cadute. La revisione a metà ciclo non ha evidenziato problemi di sicurezza significativi, e una Strategia di Valutazione e Mitigazione del Rischio (REMS) sembra non essere necessaria.
La FDA ha extendido la fecha PDUFA para la solicitud de nuevo medicamento (NDA) de Biohaven (NYSE: BHVN) para troriluzol en el tratamiento de la ataxia espinocerebelosa (SCA) por tres meses, hasta el cuarto trimestre de 2025. Esta extensión permite una revisión completa de las presentaciones recientes, y la FDA planea realizar una reunión del comité asesor. No se plantearon nuevas preocupaciones.
El troriluzol, que ha recibido la Designación Fast-Track, Medicamento Huérfano y Revisión Prioritaria por parte de la FDA, podría convertirse en el primer tratamiento aprobado por la FDA para la SCA, una rara enfermedad neurodegenerativa genética. Los datos clínicos muestran que el troriluzol, una pastilla oral de una vez al día, ralentiza la progresión de la enfermedad entre un 50 y 70% y reduce el riesgo de caídas. La revisión a mitad de ciclo no reveló preocupaciones importantes de seguridad, y parece innecesaria una Estrategia de Evaluación y Mitigación de Riesgos (REMS).
FDA가 Biohaven(NYSE: BHVN)의 트로릴루졸 신약 신청서(NDA)의 PDUFA 날짜를 3개월 연장하여 2025년 4분기로 조정했습니다. 이번 연장은 최근 제출된 자료를 완전하게 검토할 수 있도록 하며, FDA는 자문위원회 회의를 개최할 계획입니다. 새로운 우려 사항은 제기되지 않았습니다.
트로릴루졸은 FDA로부터 신속 심사(Fast-Track), 희귀의약품 지정(Orphan Drug Designation), 우선 심사(Priority Review)를 받은 약물로, 희귀 유전성 신경퇴행성 질환인 척수소뇌성 운동실조증(SCA)에 대해 FDA 최초 승인 치료제가 될 수 있습니다. 임상 데이터에 따르면 하루 한 번 복용하는 경구용 알약인 트로릴루졸은 질병 진행을 50~70% 늦추고 낙상 위험을 감소시킵니다. 중간 검토에서는 주요 안전 문제는 발견되지 않았으며, 위험 평가 및 완화 전략(REMS)은 필요하지 않은 것으로 보입니다.
La FDA a prolongé la date PDUFA pour la demande de nouveau médicament (NDA) de Biohaven (NYSE : BHVN) concernant le troriluzole pour le traitement de l'ataxie spinocérébelleuse (SCA) de trois mois, jusqu'au quatrième trimestre 2025. Cette extension permet un examen complet des soumissions récentes, et la FDA prévoit de tenir une réunion du comité consultatif. Aucune nouvelle préoccupation n’a été soulevée.
Le troriluzole, qui a reçu la désignation Fast-Track, médicament orphelin et examen prioritaire de la FDA, pourrait devenir le premier traitement approuvé par la FDA pour la SCA, une maladie neurodégénérative génétique rare. Les données cliniques montrent que le troriluzole, un comprimé oral une fois par jour, ralentit la progression de la maladie de 50 à 70% et réduit le risque de chutes. L’examen à mi-parcours n’a révélé aucun problème majeur de sécurité, et une stratégie d’évaluation et d’atténuation des risques (REMS) semble inutile.
Die FDA hat das PDUFA-Datum für den neuen Arzneimittelantrag (NDA) von Biohaven (NYSE: BHVN) für Troriluzol zur Behandlung der spinocerebellären Ataxie (SCA) um drei Monate bis zum vierten Quartal 2025 verlängert. Die Verlängerung ermöglicht eine vollständige Überprüfung der jüngsten Einreichungen, und die FDA plant eine Beratungsausschusssitzung. Es wurden keine neuen Bedenken geäußert.
Troriluzol, das von der FDA die Fast-Track-, Orphan-Drug- und Prioritätsprüfung erhalten hat, könnte die erste von der FDA zugelassene Behandlung für SCA werden, eine seltene genetische neurodegenerative Erkrankung. Klinische Daten zeigen, dass Troriluzol, eine einmal täglich einzunehmende orale Tablette, das Fortschreiten der Krankheit um 50-70% verlangsamt und das Sturzrisiko reduziert. Die Zwischenbewertung ergab keine größeren Sicherheitsbedenken, und eine Risikoevaluierungs- und Minderungsstrategie (REMS) scheint nicht erforderlich zu sein.
- Potential to be first-ever FDA-approved treatment for SCA with no current treatment options
- Clinical data shows 50-70% slowdown in disease progression
- No major safety concerns identified in mid-cycle review
- No REMS requirement anticipated, simplifying potential commercialization
- Multiple FDA designations received (Fast-Track, Orphan Drug, Priority Review)
- PDUFA date delayed by 3 months to Q4 2025
- Additional advisory committee meeting required, adding uncertainty to approval process
Insights
FDA's 3-month review extension for Biohaven's troriluzole delays potential first-ever SCA treatment approval to Q4 2025, impacting timeline but pathway remains intact.
The FDA has extended the PDUFA date for Biohaven's troriluzole by three months, pushing the decision timeline for this potential first-ever treatment for Spinocerebellar Ataxia (SCA) to Q4 2025. This extension was granted to allow for a complete review of recent submissions responding to FDA information requests, and the agency is planning an advisory committee meeting to discuss the application.
What's particularly noteworthy is that the FDA did not raise any new concerns in their communication, suggesting this is primarily a procedural delay rather than a fundamental issue with the application. The regulatory status remains favorable with troriluzole maintaining its Fast-Track designation, Orphan Drug Designation, and Priority Review status - all indicators that the FDA recognizes both the unmet medical need and the potential significance of this therapy.
The clinical profile mentioned in the release appears compelling, with data suggesting troriluzole slows disease progression by 50-70% as measured by the f-SARA scale and reduces fall risk. The completion of the mid-cycle review without identification of major safety concerns and the apparent lack of need for a Risk Evaluation and Mitigation Strategy (REMS) are additional positive regulatory signals.
This extension primarily impacts the timeline rather than the likelihood of approval, though the planned advisory committee meeting adds an element of uncertainty as these panels can be unpredictable. For a rare disease with no approved therapies, the regulatory hurdles tend to be lower when safety is established, which appears to be the case here based on the mid-cycle review results.
- Spinocerebellar Ataxia (SCA) is a rare, genetic, life-threatening neurodegenerative disease with no available treatment.
- Troriluzole has been granted Fast-Track, Orphan Drug Designation (ODD) and Priority Review from the FDA.
- Troriluzole would be the first and only FDA-approved treatment for SCA, if approved.
Biohaven previously received Fast-Track, Orphan Drug Designation (ODD) and Priority Review from the FDA regarding troriluzole for SCA. Priority Review designation is assigned to applications for drugs that would offer a significant improvement over other available treatments for a given disorder or would provide a treatment option where none exists. In the case of SCA, a rare, genetic, neurodegenerative disease, troriluzole would be the first and only FDA-approved treatment for this life-threatening disorder.
Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven stated, "SCA is a devastating neurodegenerative disease that has affected generations of families and has no current approved therapy. We are committed to bringing the first treatment to patients and families affected by SCA. The clinical data presented in the NDA show a highly favorable benefit-risk profile with troriluzole, a once-daily oral pill, slowing disease progression by 50
The Company recently announced it has completed the FDA mid-cycle review meeting and regulatory inspections of Biohaven and key clinical research sites for troriluzole in the treatment of SCA. The mid cycle review concluded that there were no previously unidentified major safety concerns, and it does not appear a Risk Evaluation and Mitigation Strategy (REMS) is needed.
About Spinocerebellar Ataxia (SCA)
Spinocerebellar ataxia is a group of dominantly inherited neurodegenerative disorders characterized by progressive loss of voluntary motor control and atrophy of the cerebellum and brainstem. SCA affects approximately 15,000 people in the United States and 24,000 in Europe and the United Kingdom. Patients experience significant morbidity, including impaired gait leading to falls, loss of ambulation and progression to a wheelchair, inability to communicate due to speech impairment, difficulty swallowing, and premature death. While signs and symptoms can appear anytime from childhood to late adulthood, SCA typically presents in early adulthood and progresses over a number of years. Currently, there are no FDA-approved treatments and no cure for SCA.
About Troriluzole
Troriluzole is a new chemical entity (NCE) and third-generation novel prodrug that modulates glutamate, the most abundant excitatory neurotransmitter in the human body. The primary mode of action of troriluzole is reducing synaptic levels of glutamate. Troriluzole increases glutamate uptake from the synapse, by augmenting the expression and function of excitatory amino acid transporters located on glial cells that play a key role in clearing glutamate from the synapse. The glutamate modulating activity of troriluzole addresses the widely documented glutamate deregulation that underlies neurodegeneration and Purkinje cell dysfunction in patients with SCA. Troriluzole also has the potential to be developed in a number of other diseases associated with excessive glutamate. More information about troriluzole can be found at the Biohaven's website: https://www.biohaven.com/pipeline/clinical-programs/glutamate/.
About Biohaven
Biohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. The company is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven's extensive clinical and preclinical programs include Kv7 ion channel modulation for epilepsy and mood disorders; extracellular protein degradation for immunological diseases; TRPM3 antagonism for migraine and neuropathic pain; TYK2/JAK1 inhibition for neuroinflammatory disorders; glutamate modulation for OCD and SCA; myostatin inhibition for neuromuscular and metabolic diseases, including SMA and obesity; antibody recruiting bispecific molecules and antibody drug conjugates for cancer. For more information, visit www.biohaven.com.
Forward-looking Statements
This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including "continue", "plan", "will", "believe", "may", "expect", "anticipate" and similar expressions, is intended to identify forward-looking statements. Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing and potential marketing approval and commercialization of development candidates, including the potential FDA approval and commercialization of troriluzole for SCA, are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various factors including: the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials; the timing of planned interactions and filings with the FDA; including those regarding the potential FDA approval of Troriluzole for SCA; the timing and outcome of expected regulatory filings; complying with applicable U.S. regulatory requirements; the potential commercialization of Biohaven's product candidates; the potential for Biohaven's product candidates to be first approved therapies; and the effectiveness and safety of Biohaven's product candidates. Additional important factors to be considered in connection with forward-looking statements are described in Biohaven's filings with the Securities and Exchange Commission, including within the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". The forward-looking statements are made as of the date of this news release, and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
Investor Contact:
Jennifer Porcelli
Vice President, Investor Relations
jennifer.porcelli@biohavenpharma.com
+1 (201) 248-0741
Media Contact:
Mike Beyer
Sam Brown Inc.
mikebeyer@sambrown.com
+1 (312) 961-2502
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SOURCE Biohaven Ltd.
FAQ
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