Chimerix Announces FDA Acceptance and Priority Review of New Drug Application for Dordaviprone as Treatment for Recurrent H3 K27M-Mutant Diffuse Glioma
Chimerix (NASDAQ: CMRX) announced FDA acceptance of its New Drug Application (NDA) for dordaviprone, targeting recurrent H3 K27M-mutant diffuse glioma treatment. The application received Priority Review status with a PDUFA target action date of August 18, 2025.
The FDA has not planned an advisory committee meeting for the application review. Dordaviprone has received Rare Pediatric Disease Designation, Fast-Track Designation in the US, and Orphan Drug Designation in the US, Europe, and Australia. The company has applied for a Rare Pediatric Disease Priority Review Voucher in the NDA submission.
If approved, dordaviprone would be the first specific treatment for patients with recurrent H3 K27M-mutant diffuse glioma, a form of high-grade glioma with treatment options beyond palliative care.
Chimerix (NASDAQ: CMRX) ha annunciato l'accettazione da parte della FDA della sua Nuova Richiesta di Farmaco (NDA) per dordaviprone, mirata al trattamento del glioma diffuso ricorrente mutante H3 K27M. La richiesta ha ricevuto lo status di Revisione Prioritaria con una data obiettivo di azione PDUFA del 18 agosto 2025.
La FDA non ha pianificato una riunione del comitato consultivo per la revisione della richiesta. Dordaviprone ha ricevuto la Designazione di Malattia Pediatrica Rara, la Designazione Fast-Track negli Stati Uniti e la Designazione di Farmaco Orfano negli Stati Uniti, in Europa e in Australia. L'azienda ha richiesto un Voucher per la Revisione Prioritaria della Malattia Pediatrica Rara nella presentazione dell'NDA.
Se approvato, dordaviprone sarebbe il primo trattamento specifico per i pazienti con glioma diffuso ricorrente mutante H3 K27M, una forma di glioma ad alto grado con opzioni di trattamento oltre la cura palliativa.
Chimerix (NASDAQ: CMRX) anunció la aceptación por parte de la FDA de su Nueva Solicitud de Medicamento (NDA) para dordaviprone, dirigido al tratamiento del glioma difuso recurrente mutante H3 K27M. La solicitud recibió el estado de Revisión Prioritaria con una fecha de acción objetivo PDUFA del 18 de agosto de 2025.
La FDA no ha programado una reunión del comité asesor para la revisión de la solicitud. Dordaviprone ha recibido la Designación de Enfermedad Pediátrica Rara, Designación de Vía Rápida en EE. UU., y Designación de Medicamento Huérfano en EE. UU., Europa y Australia. La empresa ha solicitado un Vale de Revisión Prioritaria de Enfermedad Pediátrica Rara en la presentación de la NDA.
Si se aprueba, dordaviprone sería el primer tratamiento específico para pacientes con glioma difuso recurrente mutante H3 K27M, una forma de glioma de alto grado con opciones de tratamiento más allá de la atención paliativa.
Chimerix (NASDAQ: CMRX)는 dordaviprone에 대한 새로운 약물 신청(NDA)이 FDA에 의해 수용되었다고 발표했습니다. 이 약물은 재발성 H3 K27M 변이 확산성 신경아교종 치료를 목표로 하고 있습니다. 이 신청서는 우선 심사 상태를 부여받았으며, PDUFA 목표 행동 날짜는 2025년 8월 18일입니다.
FDA는 신청서 검토를 위한 자문 위원회 회의를 계획하지 않았습니다. Dordaviprone은 희귀 소아 질환 지정, 미국에서의 신속 심사 지정 및 미국, 유럽, 호주에서의 고아 약물 지정을 받았습니다. 회사는 NDA 제출에서 희귀 소아 질환 우선 심사 바우처를 요청했습니다.
승인될 경우, dordaviprone은 재발성 H3 K27M 변이 확산성 신경아교종 환자를 위한 첫 번째 특정 치료제가 될 것입니다. 이는 완화 치료를 넘어서는 고급 신경아교종의 한 형태입니다.
Chimerix (NASDAQ: CMRX) a annoncé l'acceptation par la FDA de sa Demande de Nouveau Médicament (NDA) pour dordaviprone, visant à traiter le gliome diffus récurrent muté H3 K27M. La demande a reçu le statut de Révision Prioritaire avec une date cible d'action PDUFA du 18 août 2025.
La FDA n'a pas prévu de réunion du comité consultatif pour l'examen de la demande. Dordaviprone a reçu la désignation de Maladie Pédiatrique Rare, la désignation Fast-Track aux États-Unis et la désignation de Médicament Orphelin aux États-Unis, en Europe et en Australie. L'entreprise a demandé un Bon de Révision Prioritaire pour Maladie Pédiatrique Rares dans la soumission de la NDA.
Si approuvé, dordaviprone serait le premier traitement spécifique pour les patients atteints de gliome diffus récurrent muté H3 K27M, une forme de gliome de haut grade avec des options de traitement au-delà des soins palliatifs.
Chimerix (NASDAQ: CMRX) gab die Annahme seines Antrags auf Zulassung eines neuen Arzneimittels (NDA) für dordaviprone durch die FDA bekannt, der auf die Behandlung von wiederkehrendem H3 K27M-mutiertem diffusem Gliom abzielt. Der Antrag erhielt den Status einer Prioritätsprüfung mit einem Zieltermin für die PDUFA-Aktion am 18. August 2025.
Die FDA hat für die Überprüfung des Antrags kein Treffen des Beratungsausschusses geplant. Dordaviprone erhielt die Auszeichnung als seltene pädiatrische Erkrankung, die Fast-Track-Auszeichnung in den USA und die Orphan-Drug-Auszeichnung in den USA, Europa und Australien. Das Unternehmen hat in der NDA-Einreichung einen Voucher für die Prioritätsprüfung seltener pädiatrischer Erkrankungen beantragt.
Wenn genehmigt, wäre dordaviprone die erste spezifische Behandlung für Patienten mit wiederkehrendem H3 K27M-mutiertem diffusem Gliom, einer Form von hochgradigem Gliom mit Behandlungsoptionen über die palliative Versorgung hinaus.
- FDA granted Priority Review status for dordaviprone NDA
- Potential first-to-market treatment for recurrent H3 K27M-mutant diffuse glioma
- No advisory committee meeting planned, potentially streamlining approval process
- Multiple regulatory designations secured (Rare Pediatric, Fast-Track, Orphan Drug)
- Eligible for valuable Rare Pediatric Disease Priority Review Voucher
- Final FDA approval still pending
- Commercial launch timeline uncertain
Insights
The FDA's acceptance of Chimerix's NDA for dordaviprone marks a pivotal development with multiple strategic advantages. The absence of a planned advisory committee meeting is particularly noteworthy, as it typically indicates the FDA has no significant safety or efficacy concerns requiring external expert input. This substantially increases the probability of approval and could accelerate the timeline.
The Priority Review status carries two important implications: First, it reduces the standard 10-month review period to 6 months, potentially enabling faster market entry. Second, it maintains eligibility for a Rare Pediatric Disease Priority Review Voucher. These vouchers, which can be sold to other companies or used internally, have historically commanded prices between
The commercial opportunity is particularly compelling as dordaviprone could become the first approved treatment specifically for H3 K27M-mutant diffuse glioma. The drug's Orphan Drug Designation in multiple regions provides 7 years of market exclusivity in the US and 10 years in Europe, creating a protected market opportunity. For a rare pediatric brain tumor with treatment options beyond palliative care, this positions Chimerix to potentially capture significant market share.
The company's proactive approach to commercial preparation suggests confidence in the regulatory outcome and readiness to launch quickly post-approval. This is important for rare disease treatments where rapid market penetration and patient identification are essential for commercial success.
Potential First Approval for Patients with Recurrent H3 K27M-mutant Diffuse Glioma
PDUFA Target Action Date of August 18, 2025
No Advisory Committee Meeting Currently Planned to Discuss Application
DURHAM, N.C., Feb. 18, 2025 (GLOBE NEWSWIRE) -- Chimerix (NASDAQ: CMRX), a biopharmaceutical company whose mission it is to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases, today announced the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) seeking accelerated approval for dordaviprone as a treatment for patients with recurrent H3 K27M-mutant diffuse glioma. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of August 18, 2025. The FDA does not currently plan to hold an advisory committee meeting to discuss the application.
“This significant milestone brings us one step closer to our goal of accelerating access to the first medicine specific to patients diagnosed with recurrent H3 K27M-mutant diffuse glioma," said Mike Andriole, Chief Executive Officer of Chimerix. "Patients with this form of high-grade glioma face a very difficult prognosis with few treatment options beyond palliative care. Our team is working expeditiously with FDA to facilitate their review as we simultaneously prepare for a potential commercial launch in order to ensure rapid availability to patients in need."
Dordaviprone has received Rare Pediatric Disease Designation for H3 K27M-mutant glioma and has applied for a Rare Pediatric Disease Priority Review Voucher (PRV) in the NDA submission. The application remains eligible for this PRV by virtue of its Priority Review status. Dordaviprone also has Fast-Track Designation in the United States and Orphan Drug Designation in the United States, Europe and Australia.
About Dordaviprone
Dordaviprone (ONC201) is a novel first-in-class small molecule imipridone that selectively targets the mitochondrial protease ClpP and dopamine receptor D2 (DRD2).
About Chimerix
Chimerix is a biopharmaceutical company with a mission to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases. The Company’s most advanced clinical-stage development program, dordaviprone, is in development for H3 K27M-mutant glioma. The Company is conducting Phase 1 dose escalation studies of ONC206 to evaluate safety and PK data.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include those relating to, among other things: the possible regulatory path forward for dordaviprone, including the timing and consequences of accelerated approval, Priority Review, rare pediatric disease Priority Review vouchers and approval for marketing authorization; the timeline of related discussions with the FDA; the initial potential PDUFA timing; the timing of the U.S. commercial launch; and the expected impact of dordaviprone on patients. Among the factors and risks that could cause actual results to differ materially from those indicated in the forward-looking statements are: risks related to the ability to obtain and maintain accelerated approval, Priority Review, rare pediatric disease Priority Review vouchers, and approval for marketing authorization; completion and outcome of the Phase 3 ACTION study of dordaviprone; risks associated with market acceptance; risks associated with repeating positive results obtained in prior preclinical or clinical studies in future studies; and additional risks set forth in the Company’s filings with the Securities and Exchange Commission. These forward-looking statements represent the Company's judgment as of the date of this release. The Company disclaims, however, any intent or obligation to update these forward-looking statements.
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