FDA Action Update, February 2025: Approvals, Designations, and Acceptances

NeurologyLive. March 9, 2025. The FDA was busy in March 2025, making a number of decisions on potential new therapeutic agents including granting approvals, acceptances, and designations.

- Solid Biosciences' SGT-212 gene therapy for Friedreich ataxia received FDA clearance, targeting neurologic and systemic manifestations via dual administration routes. 

-On the same day, February 19, the FDA accepted PTC Therapeutics’ new drug application (NDA) for its investigational agent vatiquinone as a treatment for patients with Friedreich ataxia (FA)

FDA Action Update, February 2025: Approvals, Designations, and Acceptances