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 the end of 2024, seeking accelerated approval and addressing a lethal Grade 4 brain tumor with no approved therapies.

• The submission follows extensive collaboration with the FDA, disease experts, and patient advocates, and will include an application for a Rare Pediatric Disease Priority Review Voucher.

Details of approval or decision

• The NDA will seek accelerated approval, priority review, and multiple FDA designations, including Orphan Drug, Fast Track, and Rare Pediatric Disease, with a potential PDUFA action date and commercial launch in Q3 2025 if granted.

• Dordaviprone has rare pediatric disease designation, making it eligible for a priority review voucher.

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

Impact on industry and stakeholders

• If approved, dordaviprone would be the first FDA-approved therapy for H3K27M-mutant glioma, addressing a critical unmet need for over 2,000 US patients annually, primarily children and young adults.

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

• Commercial preparations include hiring industry-leading talent, developing patient support services, and engaging with neuro-oncology centers of excellence.