The U.S. Food and Drug Administration (FDA) has approved Waskyra (etuvetidigene autotemcel) for the treatment of patients ages 6 months and older with Wiskott-Aldrich syndrome (WAS).
WAS is a disease with immunological deficiency and reduced ability to form blood clots. Signs and symptoms include easy bruising or bleeding, susceptibility to infections and to immune and inflammatory disorders, and an increased risk for some cancers. Eczema is also common in people with WAS. WAS is caused by genetic changes in the WAS gene.
Etuvetidigene autotemcel is an ex-vivo gene therapy that consists of the patient’s own hematopoietic stem cells being genetically modified to include functional copies of the WAS gene. Following reduced-intensity conditioning, the gene-corrected cells are infused intravenously to restore blood cell production and functional WAS protein expression in affected cells, addressing the underlying cause of the disease.
The approval is based on data from two open-label, single-arm, multinational clinical trials and an expanded access program. In 27 patients, etuvetidigene autotemcel helped reduce the rate of severe infections by 93% in the first six to 18 months, and moderate and severe bleeding by 60% in the first 12 months after treatment. Most patients did not report moderate to severe bleeding after four years post treatment.
The most common side effects associated with the therapy include rash, respiratory tract infection, vomiting, and diarrhea.
This therapy was submitted and developed by a non-profit organization, Fondazione Telethon, based in Italy.
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To learn more about WAS and other rare autoimmune conditions, visit https://checkrare.com/diseases/autoimmune-and-auto-inflammatory-disorders/
