FDA Flexibility Leads to First Approved Gene Therapy for Rare Blood & Immune System Disorder

A genetic disorder that leads to bleeding episodes and life-threatening infections has a new FDA approved treatment, a regulatory decision that marks two notable firsts — the first gene therapy for this rare disease, Wiskott-Aldrich syndrome, and the first cell and gene therapy approval awarded to a non-profit entity.

The FDA decision announced late Tuesday covers the treatment of children age six months and older who have the mutation driving Wiskott-Aldrich syndrome. These patients must also be eligible for a stem cell transplant but cannot get one because no matched donor is available. This gene therapy from Fondazione Telethon, known in development as etuvetidigene autotemcel (etu-cel for short), will be commercialized under the brand name Waskyra. The therapy is also making regulat