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.
Vertex agreed to purchase Crinetics Pharmaceuticals for $85 per share in cash, a strategic move to diversify its portfolio beyond cystic fibrosis treatments.
Read the full storyThe FDA granted Rare Pediatric Disease Designation to Vanda's VCA-894A, potentially fast-tracking the development of its genetic neurological therapy.
The FDA accepted Agios' sNDA for mitapivat with a Priority Review designation, setting a PDUFA goal date of November 1, 2026.
Vertex Pharmaceuticals secures regulatory expansion for its CRISPR-based therapy Casgevy to treat pediatric patients as young as two years old.