Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a non-profit sponsor to win FDA approval.
The FDA has approved the gene therapy etuvetidigene autotemcel for the treatment of Wiskott-Aldrich syndrome, a rare genetic disorder characterized by immune system abnormalities. The therapy will carry the brand name Waskyra.
Waskyra is the first gene therapy for this indication, according to the FDA’s news release on Tuesday—and the first approved genetic treatment from a non-profit sponsor. Waskyra was sponsored by Fondazione Telethon ETS, an Italy-based organization that funds research into and the development of therapies for rare diseases.
With the FDA’s clearance, Waskyra can now be used in patients six months and older harboring a mutation in the WAS gene who are eligible for hematopoietic stem cell transplantation but have no suitable matched donor. FDA Commissioner Marty Makary called Waskyra’s approval a “transformative milestone” for patients living with Wiskott-Aldrich syndrome.
Affecting between one and 10 males per million globally, Wiskott-Aldrich syndrome is caused by mutations to the WAS gene, which under healthy circumstances produces a protein that facilitates white blood cell function and platelet maturation. As a result, patients suffer from frequent and prolonged bleeding episodes, heightened risk of infections and a greater likelihood of developing autoimmune disorders and cancer. The disease is even rarer in females.
Waskyra is a cell-based therapy that makes use of a patient’s own hematopoietic stem cells. The cells are altered outside the body to carry functional copies of the WAS gene before being reinfused into the patient. In turn, Waskyra treatment restores healthy production of the WAS protein.
The FDA’s verdict on Tuesday was backed by data from two open-label, single-arm studies covering a total of 27 patients with severe disease. Results showed that severe infections dropped 93% from six to 18 months after treatment, compared to 12 months before Waskyra dosing. Patients given the gene therapy also saw a 60% reduction in moderate and severe bleeding events.
These findings, the agency noted, pointed to Waskyra’s “substantial and sustained clinical benefit,” with “significant reductions in the primary disease manifestations.”
As for safety, Waskyra’s common side effects included rashes, respiratory infection and catheter-related infections.
