The US Food and Drug Administration said on Tuesday it had approved the first gene therapy for a rare and life-threatening immune disorder.
The therapy, Waskyra, was approved for Wiskott-Aldrich syndrome (WAS), which weakens the immune system, making patients prone to frequent infections, easy bleeding and bruising, and skin problems such as eczema.
Waskyra, also known as etuvetidigene autotemcel, is indicated for patients aged 6 months and older and adults with a WAS gene mutation.
Approval was granted to the Italian non-profit Fondazione Telethon ETS.
It uses the patient's own blood stem cells, which are genetically modified to include functional copies of the WAS gene, the FDA said.
Gene therapy approval effects
The approval was based on two open-label studies and an expanded access program, totaling 27 patients, in which the therapy reduced the rate of severe infections by 93% during the first 6 to 18 months and moderate and severe bleeding by 60% during the first 12 months after treatment, the agency said.
The most common side effects associated with Waskyra include rash, respiratory tract infection, vomiting, and diarrhea.
"The FDA continues to exercise flexibility in the regulatory approach for rare diseases by considering all available data sources, including, as appropriate, data from expanded access programs," said FDA chief medical and scientific officer Vinay Prasad.