FDA Approves First Nonprofit-Led Gene Therapy for Wiskott-Aldrich Syndrome

A gene therapy developed by an Italian nonprofit, Fondazione Telethon, known as Waskyra (etuvetidigene autotemcel), has received U.S. Food and Drug Administration approval for treating Wiskott-Aldrich syndrome, a rare immune disorder with limited treatment options. The decision marks the first time the FDA has authorized a gene therapy from a nonprofit applicant and reflects a flexible regulatory approach that considered diverse clinical data from small open-label studies and expanded access programs.

Endpoints News

+1

👉 Read the full article: In a first, FDA approves a gene therapy from a nonprofit

Disclaimer

Images are illustrative only. No medical advice is provided. SSCTR is not responsible for clinical or regulatory decisions. Content is informational and educational.