The first FDA-approved gene therapy for a neuromuscular disease in the United States is a wonderful hope for patients, also a key challenge for France

On May 24, the Food and Drug Administration approved Zolgensma®, a gene therapy drug for the treatment of spinal muscular atrophy developed by AveXis (Novartis).

AFM-Telethon welcomes this historic step for patients and families affected by this neuromuscular disease, whose most severe form kills toddlers before the age of two. AFM-Telethon urges the European Agency (EMA) to grant quickly the approval of Zolgensma® for Europe. In the meantime, it asks that French patients are being treated as part of the patient access measures available in France (temporary authorization of use).
This historic step confirms AFM- Téléthon’s visionary strategy and choices, in driving, supporting and financing the development of gene therapy for nearly 30 years. It also confirms the key role played by its laboratory Genethon, created thanks to the Telethon. Following the discovery of the gene responsible for SMA in 1995 with AFM-Telethon fundings, Genethon research has been decisive in the design of both the product and the route of administration of this first gene therapy for a neuromuscular disease, thanks to the pioneering work of Martine Barkats and her team.