FDA Advisory Committee Votes Favorably that the Data Support Arimoclomol as Effective Treatment for Patients with Niemann-Pick Disease Type C
Zevra Therapeutics (NasdaqGS: ZVRA) announced that the FDA's Genetic Metabolic Diseases Advisory Committee voted 11-5 in favor of arimoclomol's effectiveness in treating Niemann-Pick disease type C (NPC). The committee's recommendation will be considered by the FDA as it reviews the arimoclomol New Drug Application (NDA), with a PDUFA action date of September 21, 2024. Zevra's CEO, Neil F. McFarlane, expressed confidence in arimoclomol's clinical benefit based on data from the pivotal trial, long-term open-label extension study, and expanded access programs. The GeMDAC's decision, while not binding, is a positive step towards potential FDA approval for this rare disease treatment.
Zevra Therapeutics (NasdaqGS: ZVRA) ha annunciato che il Comitato Consultivo sui Disturbi Metabolici Genetici della FDA ha votato 11-5 a favore dell'efficacia dell'arimoclomolo nel trattamento della malattia di Niemann-Pick di tipo C (NPC). La raccomandazione del comitato sarà presa in considerazione dalla FDA mentre esamina la Domanda di Nuovo Farmaco (NDA) per l'arimoclomolo, con una data di scadenza PDUFA del 21 settembre 2024. Il CEO di Zevra, Neil F. McFarlane, ha espresso fiducia nei benefici clinici dell'arimoclomolo basati su dati provenienti dallo studio clinico pivotal, dallo studio di estensione a lungo termine in aperto e dai programmi di accesso espanso. La decisione del GeMDAC, sebbene non vincolante, rappresenta un passo positivo verso una possibile approvazione della FDA per questo trattamento di una malattia rara.
Zevra Therapeutics (NasdaqGS: ZVRA) anunció que el Comité Asesor de Enfermedades Metabólicas Genéticas de la FDA votó 11-5 a favor de la efectividad del arimoclomol para tratar la enfermedad de Niemann-Pick tipo C (NPC). La recomendación del comité será considerada por la FDA mientras revisa la Solicitud de Nuevo Medicamento (NDA) para el arimoclomol, con una fecha de acción PDUFA del 21 de septiembre de 2024. El CEO de Zevra, Neil F. McFarlane, expresó confianza en el beneficio clínico del arimoclomol basado en datos del ensayo clínico pivotal, del estudio de extensión a largo plazo y de los programas de acceso expandido. La decisión del GeMDAC, aunque no vinculante, es un paso positivo hacia la posible aprobación de la FDA para este tratamiento de una enfermedad rara.
Zevra Therapeutics (NasdaqGS: ZVRA)는 FDA의 유전 대사 질환 자문 위원회가 Niemann-Pick 병형 C (NPC) 치료에 대한 arimoclomol의 효과에 대해 11-5로 찬성 투표했다고 발표했습니다. 위원회의 권고는 FDA가 arimoclomol의 신약 신청(NDA)을 검토하는 동안 고려될 것이며, PDUFA 시행 날짜는 2024년 9월 21일입니다. Zevra의 CEO인 Neil F. McFarlane은 결정적인 시험, 장기 개방형 연장 연구 및 확대 접근 프로그램에서 얻은 데이터를 바탕으로 arimoclomol의 임상적 이점에 대한 신뢰를 표명했습니다. GeMDAC의 결정은 구속력이 없지만 이 희귀 질환 치료에 대한 FDA 승인을 향한 긍정적인 발전입니다.
Zevra Therapeutics (NasdaqGS: ZVRA) a annoncé que le Comité Consultatif des Maladies Métaboliques Génétique de la FDA a voté 11-5 en faveur de l'efficacité de l'arimoclomol dans le traitement de la maladie de Niemann-Pick de type C (NPC). La recommandation du comité sera prise en compte par la FDA lors de l'examen de la Demande de Nouveau Médicament (NDA) pour l'arimoclomol, avec une date d'action PDUFA fixée au 21 septembre 2024. Le PDG de Zevra, Neil F. McFarlane, a exprimé sa confiance dans le bénéfice clinique de l'arimoclomol basé sur des données issues de l'essai clinique pivot, de l'étude d'extension à long terme et des programmes d'accès élargi. La décision du GeMDAC, bien qu'elle ne soit pas contraignante, constitue un pas positif vers l'approbation potentielle de la FDA pour ce traitement d'une maladie rare.
Zevra Therapeutics (NasdaqGS: ZVRA) gab bekannt, dass das genetisch-metabolische Erkrankungen beratende Komitee der FDA mit 11-5 zugunsten der Wirksamkeit von Arimoclomol zur Behandlung der Niemann-Pick-Krankheit Typ C (NPC) gestimmt hat. Die Empfehlung des Komitees wird von der FDA berücksichtigt, während sie den Antrag auf Zulassung eines neuen Arzneimittels (NDA) für Arimoclomol prüft, mit einem PDUFA-Fälligkeitsdatum vom 21. September 2024. Der CEO von Zevra, Neil F. McFarlane, äußerte Vertrauen in den klinischen Nutzen von Arimoclomol, basierend auf Daten aus der entscheidenden Studie, der langfristigen offenen Verlängerungsstudie und den Programmen für erweiterten Zugang. Die Entscheidung des GeMDAC ist zwar nicht verbindlich, stellt jedoch einen positiven Schritt in Richtung einer möglichen FDA-Zulassung für diese Behandlung seltener Krankheiten dar.
- FDA Advisory Committee voted favorably (11-5) on arimoclomol's effectiveness for NPC
- Positive recommendation from GeMDAC increases likelihood of FDA approval
- Comprehensive clinical data supports arimoclomol's efficacy in treating NPC
- FDA's final decision is still pending, with PDUFA date set for September 21, 2024
- Advisory Committee's recommendation is not binding on the FDA
Insights
This FDA advisory committee vote is a significant milestone for Zevra Therapeutics and patients with Niemann-Pick Disease Type C (NPC). The 11-5 favorable vote suggests strong support for arimoclomol's efficacy, which could pave the way for FDA approval. However, it's important to note that while positive, this vote is not binding on the FDA's final decision.
The committee's decision was based on comprehensive data, including the pivotal trial, long-term open-label extension study and expanded access programs. This breadth of evidence strengthens the case for arimoclomol's clinical benefit. The
This favorable FDA advisory committee vote is a positive catalyst for Zevra Therapeutics (NASDAQ: ZVRA). If approved, arimoclomol could become a significant revenue driver for the company, given the rarity of NPC and potential for orphan drug pricing. However, investors should be cautious as final FDA approval is not guaranteed.
The market for rare disease treatments is lucrative, with high unmet needs and potential for premium pricing. A successful launch could significantly boost Zevra's financial outlook. Investors should watch for the PDUFA date of September 21, 2024, as it will be a important inflection point for the stock. In the meantime, any updates on manufacturing readiness or commercialization plans could provide insights into the company's preparedness for a potential launch.
The favorable GeMDAC vote is a positive signal in the regulatory process for arimoclomol, but it's important to remember that the FDA is not bound by this recommendation. The
The FDA will now conduct its own independent review, considering the totality of evidence presented. Key factors will likely include the safety profile, efficacy data consistency and manufacturing quality. The involvement of patient advocacy groups and independent experts in the process adds weight to the patient perspective, which is increasingly important in rare disease drug approvals. As we approach the PDUFA date of September 21, 2024, watch for any additional information requests or communications from the FDA, which could provide clues about the likelihood of approval.
PDUFA Action Date for the Arimoclomol NDA is September 21, 2024
CELEBRATION, Fla., Aug. 02, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favorably (11 yes, 5 no) that the data support that arimoclomol is effective in the treatment of patients with Niemann-Pick disease type C (NPC).
“We are extremely pleased with the committee’s recognition of the benefits of arimoclomol for people living with NPC,” said Neil F. McFarlane, President and Chief Executive Officer of Zevra. “Based on the totality of the clinical data, including data from the pivotal trial, the long-term data from the arimoclomol open label extension study, and data from our expanded access programs (EAP: NCT04316637), we remain confident in the clinical benefit offered by arimoclomol as a treatment for NPC, and are optimistic about its continued path to approval.”
The GeMDAC, which consists of experts in the fields of medical genetics, inborn errors of metabolism, epidemiology and other related specialties, discussed the benefits and risks of arimoclomol, including the data recently presented at the 45th Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD), and reviewed comments received from independent experts, NPC patients, and patient advocacy group representatives. The committee’s recommendation will be considered by the FDA as it completes its independent review of the arimoclomol NDA; however, the feedback from the GeMDAC is not binding upon the Agency. The arimoclomol NDA has been assigned a Prescription Drug User Fee Act (PDUFA) action date of September 21, 2024.
About Niemann-Pick Disease Type C (NPC):
Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various tissue areas, including brain tissue. The disease is caused by mutations in the NPC1 or NPC2 genes, which are responsible for making lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Disease progression is irreversible and can be fatal within months or take years to be diagnosed and advance in severity.
About Arimoclomol:
Arimoclomol, Zevra’s orally-delivered, investigational drug product candidate for the treatment of NPC, has been granted Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, and Rare Pediatric Disease designation by the FDA, and Orphan Medicinal Product designation for the treatment of NPC by the European Medicines Agency (EMA). The FDA has accepted the resubmission of the NDA for arimoclomol and has set a user fee goal action date (PDUFA date) of September 21, 2024.
About Zevra Therapeutics:
Zevra Therapeutics is a rare disease company combining science, data, and patient needs to create transformational therapies for diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community.
For more information, please visit www.zevra.com or follow us on X (formerly Twitter) and LinkedIn.
Cautionary Note Concerning Forward-Looking Statements:
This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding the promise and potential impact of our preclinical or clinical trial data; the potential benefits of any of our products or product candidates for any specific disease or at any dosage; the impact of meetings or communications with the FDA or any advisory committee; decisions by the FDA or any other entity for arimoclomol or any other product candidates; our strategic and product development objectives, including with respect to becoming a leading, commercially focused rare disease company; and the timing of any of the foregoing. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, and other important factors that may cause our 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 and other important factors are described in detail in the "Risk Factors" section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2023, Zevra’s quarterly report for the three months ended March 31, 2024, and Zevra’s other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release.
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