Chimerix (CMRX) FDA Announcement summary

Introduction and purpose

• Plans are underway to submit a New Drug Application (NDA) for dordaviprone for recurrent H3K27M-mutant diffuse glioma in the US before year-end 2024, seeking accelerated approval for this invariably lethal disease, especially affecting children and young adults.

• The NDA will include an application for a Rare Pediatric Disease Priority Review Voucher and is supported by recent program milestones and substantial clinical data.

• The company has engaged closely with the FDA, disease experts, and patient advocates to advance the regulatory process.

Details of approval or decision

• NDA submission is planned for December 2024, with a request for Priority Review and multiple FDA designations, including Orphan Drug, Fast Track, and Rare Pediatric Disease.

• If accepted and granted Priority Review, the potential PDUFA action date is expected in Q3 2025.

• The drug has Rare Pediatric Disease Designation, making it eligible for a Priority Review Voucher.

• Plans for provisional approval in Australia are deferred, with focus on the US submission.

Impact on industry and stakeholders

• Approval would mark the first FDA-approved therapy specific to H3K27M-mutant diffuse glioma, addressing a significant unmet need for over 2,000 patients annually in the US, primarily children and young adults.

• The total addressable US market exceeds $1 billion, with ultra-orphan drug pricing anticipated.

• The launch strategy includes a nimble sales force, robust patient support, and engagement with neuro-oncology centers of excellence, aiming for readiness by Q3 2025.

• High awareness and enthusiasm among neuro-oncology professionals and patient advocates, as shown in recent market research and conferences.