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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