FDA grants PM359 Regenerative Medicine Advanced Therapy designation for chronic granulomatous disease

Prime Medicine/PM359 (CGD)

The FDA granted PM359 Regenerative Medicine Advanced Therapy (RMAT) designation, providing closer FDA guidance and possible accelerated review, based on NEJM-published Phase 1/2 results in chronic granulomatous disease. PM359 targets the p47phox form of CGD, which makes up about a quarter of cases and has no approved gene-editing treatment, with a BLA submission planned for the first half of 2027.

• RMAT designation allows intensive FDA guidance and expedited review pathway
• Targets p47phox-deficient chronic granulomatous disease; ~25% of CGD cases
• Phase 1/2 data previously published in NEJM; no approved gene-editing treatment exists for this form
• Also holds Fast Track, Orphan Drug, and Rare Pediatric Disease designations; BLA submission planned H1 2027