Chimerix, a biopharmaceutical company focused on developing treatments for life-threatening diseases, announced a significant step forward in its mission with the FDA's acceptance of its New Drug Application (NDA) for dordaviprone. The application seeks accelerated approval for the drug as a treatment for recurrent H3 K27M-mutant diffuse glioma, a rare and aggressive form of brain cancer.
The FDA has granted the application a Priority Review designation, which is given to drugs that have the potential to provide a significant improvement in the safety or effectiveness of the treatment of a serious condition. This designation expedites the review process, setting a PDUFA (Prescription Drug User Fee Act) target action date of August 18, 2025. The FDA has also indicated that it does not currently plan to convene an advisory committee meeting to discuss the application, which can often streamline the review timeline.
This development is particularly notable because if approved, dordaviprone could become the first medicine specifically for patients with recurrent H3 K27M-mutant diffuse glioma. As noted by Chimerix's CEO, Mike Andriole, patients with this form of high-grade glioma face a very difficult prognosis with limited treatment options beyond palliative care. The company is working closely with the FDA to facilitate the review and is simultaneously preparing for a potential commercial launch to ensure rapid availability of the drug to patients in need.
Dordaviprone (also known as ONC201) is a novel, first-in-class small molecule that works by selectively targeting the mitochondrial protease ClpP and dopamine receptor D2 (DRD2).
The drug has received several key designations from regulatory bodies, highlighting its potential importance. In the United States, it has been granted Rare Pediatric Disease Designation for H3 K27M-mutant glioma, making it eligible for a Rare Pediatric Disease Priority Review Voucher (PRV) upon approval. It also holds Fast-Track Designation in the U.S. and Orphan Drug Designation in the U.S., Europe, and Australia. These designations are intended to encourage the development of drugs for serious or life-threatening diseases that affect small populations.
The announcement marks a crucial milestone for Chimerix as it moves closer to its goal of providing a targeted treatment for a patient population with a significant unmet medical need. The company's press release underscores its commitment to not only the development but also the swift delivery of this potential new therapy.