Chimerix to Submit Dordaviprone for Accelerated Approval to U.S. FDA for Patients with Recurrent H3 K27M-Mutant Diffuse Glioma Before Year-End
Chimerix (CMRX) announces plans to submit a New Drug Application (NDA) for dordaviprone to the FDA before year-end, seeking accelerated approval for treating recurrent H3 K27M-mutant diffuse glioma. The company anticipates potential approval by Q3 2025, pending Priority Review.
The submission follows productive FDA interactions and includes key data showing a 28% objective response rate, 10.4-month median duration of response, and 4.6-month median time to response under RANO 2.0 criteria. Dordaviprone could become the first FDA-approved therapy for this rare disease affecting over 2,000 U.S. patients annually.
The drug has received Rare Pediatric Disease Designation and is eligible for a Priority Review Voucher. Chimerix is preparing for potential U.S. launch as early as Q3 2025.
Chimerix (CMRX) annuncia piani per presentare una Nuova Domanda di Farmaco (NDA) alla FDA entro la fine dell'anno, cercando l'approvazione accelerata per il trattamento del glioma diffuso ricorrente mutato H3 K27M. L'azienda prevede un possibile approvazione entro il terzo trimestre del 2025, in attesa di una Revisione Prioritaria.
La presentazione segue interazioni produttive con la FDA e include dati chiave che mostrano un tasso di risposta obiettivo del 28%, una durata mediana della risposta di 10,4 mesi e un tempo mediano di risposta di 4,6 mesi secondo i criteri RANO 2.0. Dordaviprone potrebbe diventare la prima terapia approvata dalla FDA per questa malattia rara che colpisce oltre 2.000 pazienti negli Stati Uniti ogni anno.
Il farmaco ha ricevuto la Designazione di Malattia Pediatrica Rara ed è idoneo per un Voucher di Revisione Prioritaria. Chimerix si sta preparando per un possibile lancio negli Stati Uniti già entro il terzo trimestre del 2025.
Chimerix (CMRX) anuncia planes para presentar una Nueva Solicitud de Medicamento (NDA) a la FDA antes de fin de año, buscando la aprobación acelerada para el tratamiento del glioma difuso recurrente mutado H3 K27M. La compañía anticipa una posible aprobación para el tercer trimestre de 2025, sujeto a una Revisión Prioritaria.
La presentación sigue interacciones productivas con la FDA e incluye datos clave que muestran una tasa de respuesta objetiva del 28%, una duración mediana de la respuesta de 10.4 meses y un tiempo mediano hasta la respuesta de 4.6 meses bajo los criterios RANO 2.0. Dordaviprone podría convertirse en la primera terapia aprobada por la FDA para esta enfermedad rara que afecta a más de 2,000 pacientes en EE. UU. anualmente.
El medicamento ha recibido la Designación de Enfermedad Pediátrica Rara y es elegible para un Voucher de Revisión Prioritaria. Chimerix se está preparando para un posible lanzamiento en EE. UU. tan pronto como en el tercer trimestre de 2025.
Chimerix (CMRX)는 연말까지 FDA에 도르다비프론에 대한 신약 신청(NDA)을 제출할 계획을 발표했으며, H3 K27M 변이 확산 신경교종 치료를 위한 가속 승인을 요청하고 있습니다. 회사는 우선 심사를 거쳐 2025년 3분기 내에 승인될 것으로 예상하고 있습니다.
이번 제출은 FDA와의 생산적인 상호작용을 바탕으로 하며, 객관적 반응률 28%, 반응 지속 시간 10.4개월, RANO 2.0 기준에 따른 반응까지의 중간 시간 4.6개월을 나타내는 주요 데이터를 포함하고 있습니다. 도르다비프론은 매년 2,000명 이상의 미국 환자에게 영향을 미치는 이 희귀 질병에 대해 FDA가 승인한 첫 번째 치료제가 될 수 있습니다.
이 약물은 희귀 소아 질환 지정(BPD designation)을 받아 우선 심사 바우처를 받을 수 있는 자격을 갖추고 있습니다. Chimerix는 2025년 3분기 내에 미국 시장 출시를 준비하고 있습니다.
Chimerix (CMRX) annonce des projets de soumettre une Demande de Nouveau Médicament (NDA) à la FDA avant la fin de l'année, en recherchant une approbation accélérée pour le traitement du gliome diffus récurrent mutant H3 K27M. L'entreprise anticipe une approbation potentielle d'ici le troisième trimestre 2025, sous réserve d'un examen prioritaire.
La soumission fait suite à des interactions productives avec la FDA et comprend des données clés montrant un taux de réponse objectif de 28%, une durée médiane de réponse de 10,4 mois et un délai médian de réponse de 4,6 mois selon les critères RANO 2.0. Dordaviprone pourrait devenir la première thérapie approuvée par la FDA pour cette maladie rare qui affecte plus de 2 000 patients aux États-Unis chaque année.
Le médicament a reçu la désignation de maladie pédiatrique rare et est éligible à un bon de révision prioritaire. Chimerix se prépare à un lancement potentiel aux États-Unis dès le troisième trimestre 2025.
Chimerix (CMRX) gibt bekannt, dass plans für die Einreichung eines Neuen Arzneimittelantrags (NDA) bei der FDA bis Ende des Jahres bestehen, um eine beschleunigte Genehmigung für die Behandlung von rezidivierenden H3 K27M-mutierten diffusen Gliomen zu beantragen. Das Unternehmen erwartet eine mögliche Genehmigung bis zum dritten Quartal 2025, vorbehaltlich einer prioritären Überprüfung.
Die Einreichung folgt produktiven Interaktionen mit der FDA und enthält wichtige Daten, die eine objektive Ansprechrate von 28 %, eine mediane Ansprechdauer von 10,4 Monaten sowie eine mediane Zeit bis zur Antwort von 4,6 Monaten nach den RANO 2.0-Kriterien zeigen. Dordaviprone könnte die erste von der FDA zugelassene Therapie für diese seltene Erkrankung werden, die jährlich über 2.000 Patienten in den USA betrifft.
Das Medikament hat die Auszeichnung als Seltene Pädiatrische Erkrankung erhalten und ist für einen Prioritätsüberprüfungs-Voucher berechtigt. Chimerix bereitet sich auf eine mögliche Einführung in den USA bereits im dritten Quartal 2025 vor.
- First potential FDA-approved therapy for H3 K27M-mutant glioma
- Demonstrated 28% objective response rate in Phase 2 trials
- 10.4-month median duration of response
- Eligible for Priority Review and Rare Pediatric Disease Priority Review Voucher
- Addresses market of 2,000+ patients annually in US
- Extended timeline to potential approval (Q3 2025)
- Still pending FDA acceptance and Priority Review approval
- Accelerated approval pathway indicates additional confirmatory trials may be needed
Insights
Potential Approval in Q3 2025 in Recurrent H3 K27M-Mutant Diffuse Glioma
Submission Plan Follows Productive and Collaborative Pre-NDA Interactions with FDA
Company to Host Conference Call on Tuesday, December 10 at 8:30 AM ET
DURHAM, N.C., Dec. 09, 2024 (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 that, following extensive dialogue with the U.S. Food and Drug Administration (FDA), the Company plans to submit a complete New Drug Application (NDA) seeking accelerated approval for dordaviprone as a treatment for recurrent H3 K27M-mutant diffuse glioma in the United States before year-end.
“We expect that, if approved, dordaviprone will fundamentally change the treatment landscape for patients suffering from this lethal form of brain cancer who have extremely limited treatment options. We have worked collaboratively with the U.S. FDA, disease experts and patient advocates throughout the year to potentially accelerate access to dordaviprone for this patient community,” said Mike Andriole, Chief Executive Officer of Chimerix. “In anticipation of a potential approval, we have bolstered our commercial leadership team and will be ready for a U.S. launch as early as the third quarter of 2025, pending application acceptance and Priority Review, if granted.”
“As a pediatric oncologist, this program is particularly meaningful given the impact a potential approval would have on children and young adults devastated by this disease. We are confident that the data generated to date could support an accelerated approval for this urgent unmet medical need,” said Allen Melemed M.D., Chief Medical Officer of Chimerix. “H3 K27M mutant gliomas are extremely aggressive and affect over 2,000 patients annually in the United States. If successful, dordaviprone would be the first FDA-approved therapy for this lethal disease, as well as one of the first molecularly defined approvals for any high-grade glioma.”
The following recent program milestones and additional supportive data were extensively discussed with the FDA and will be included in the NDA:
- Substantial enrollment of the Phase 3 ACTION study
- Phase 2 objective response rate of the 50-patient primary efficacy analysis assessed by blinded independent central review (BICR) as the primary basis of efficacy in the NDA
- Several response assessments, including the most contemporary response assessment criteria for gliomas, Response Assessment in Neuro-Oncology 2.0 (RANO 2.0), under which dordaviprone demonstrated an objective response rate of
28% , a median duration of response of 10.4 months and a median time to response of 4.6 months - Additional clinical data sets and patient narratives supportive of the primary efficacy analysis
- Clinical and nonclinical demonstration of dordaviprone-driven reversal of the central hallmark of H3 K27M-mutant glioma, H3K27 trimethyl loss
- Comprehensive safety database of glioma patients and healthy volunteers that supports a favorable benefit/risk profile
- Comprehensive clinical pharmacology and chemistry, manufacturing, and controls (CMC) studies
Chimerix will request Priority Review for the NDA. If granted, the resulting six-month FDA review period is expected to result in a potential initial Prescription Drug User Fee Act (PDUFA) action date in the third quarter of 2025.
Dordaviprone has received Rare Pediatric Disease Designation for H3 K27M-mutant glioma and is eligible to apply for a Rare Pediatric Disease Priority Review Voucher (PRV). Chimerix intends to apply for a Rare Pediatric Disease PRV in the upcoming NDA submission.
Conference Call and Webcast
Chimerix will host a conference call and live audio webcast on Tuesday, December 10 at 8:30 a.m. ET. To access the live conference call, please dial 646-307-1963 (domestic) or 800-715-9871 (international) at least five minutes prior to the start time and refer to conference ID 8870160. A live audio webcast of the call will also be available on the Investors section of Chimerix’s website, www.chimerix.com. An archived webcast will be available on the Chimerix website approximately two hours after the event.
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.
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).
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 potential to seek accelerated approval, Priority Review, rare pediatric disease Priority Review vouchers and approval for marketing authorization; timing and consequences of an NDA submission to FDA; FDA’s acceptance for filings; the timeline of related discussions with the FDA; the initial potential PDUFA timing; the potential commercial opportunity; the ability of dordaviprone to attain significant market acceptance among disease experts, patient advocates and their patients; 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; uncertainty on the response of regulators to including additional supportive data to be submitted in the NDA filing, including RANO 2.0 assessments, and uncertainty with respect to the initial potential PDUFA timing; risks related to the timing, 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; risks related to the clinical development of our clinical candidates; 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.
CONTACT:
Will O’Connor
Stern Investor Relations
212-362-1200
Dana Davis
Steelwire Public Relations
dana@steelwire.co
FAQ
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