Wedoany.com Report-Dec.10, The United States Food and Drug Administration (FDA) announced on Tuesday the approval of Waskyra (etuvetidigene autotemcel), the first gene therapy for Wiskott-Aldrich syndrome (WAS), a rare and serious inherited immune disorder.

Waskyra is indicated for patients aged six months and older, including adults, who carry mutations in the WAS gene. The condition severely impairs immune function, leading to recurrent severe infections, bleeding tendencies, bruising, and skin conditions such as eczema.

Developed by the Italian non-profit Fondazione Telethon ETS, Waskyra is an autologous therapy that uses the patient's own hematopoietic stem cells. These cells are collected, genetically modified ex vivo to insert functional copies of the WAS gene, and then reinfused into the patient after conditioning treatment.

Approval was supported by clinical data from two open-label studies and an expanded access programme involving a total of 27 patients. Results demonstrated a 93 percent reduction in severe infections within six to 18 months post-treatment and a 60 percent decrease in moderate-to-severe bleeding episodes within the first 12 months.

Common side effects observed include rash, respiratory tract infections, vomiting, and diarrhoea.

"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 Vinay Prasad, the FDA's chief medical and scientific officer.

Waskyra represents a significant advance in the treatment of Wiskott-Aldrich syndrome, offering a one-time therapy that addresses the underlying genetic cause rather than managing symptoms. The approval expands therapeutic options for patients with this life-threatening condition and highlights continued progress in gene therapy for rare inherited disorders.