Vertex Receives Expanded Approval for Casgevy Gene Therapy in Younger Children

Event Summary

On July 1, Vertex received expanded approval for its Casgevy therapy to include children as young as two years old. On July 7, the U.S. FDA accepted the supplemental New Drug Application (sNDA) for mitapivat in sickle cell disease with Priority Review. The FDA set a PDUFA goal date of November 1, 2026, for the approval decision. Additionally, the FDA granted Rare Pediatric Disease Designation to VCA-894A, an experimental therapy for Charcot-Marie-Tooth disease type 2S.