Prime Medicine (NASDAQ: PRME) has announced that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to PM359, an investigational autologous Prime Edited hematopoietic stem cell therapy for the treatment of p47phox-deficient chronic granulomatous disease (CGD).
According to Prime, RMAT designation was granted based on Phase 1/2 clinical data, including data previously published in The New England Journal of Medicine, and will provide the benefits of intensive FDA guidance and expedited review through the program’s development.
In a statement, Allan Reine, MD, CEO of Prime, commented, “FDA’s decision to grant RMAT designation to PM359 reinforces the potential for this program to deliver a meaningful, disease-modifying impact in CGD, where patients face significant morbidity, lifelong complications, and limited treatment options. The combination of RMAT, Fast Track, Orphan Drug, and Rare Pediatric Disease Designations, underscores the seriousness of CGD and the need for transformative therapies that deliver durable benefit, while positioning us to engage with the FDA on the most efficient path to a Biologics License Application. We are working with urgency to advance PM359 toward potential approval, while building on this foundation across our broader pipeline, including programs in Wilson Disease and Alpha-1 Antitrypsin Deficiency.”
