Drugs

FDA OKs 1st Gene Therapy for Wiskott-Aldrich Syndrome

FDA has approved Italian non-profit Fondazione Telethon’s BLA for Waskyra (etuvetidigene autotemcel), the first gene therapy for Wiskott-Aldrich syndrome (WAS), a rare, life-threatening immune disorder. Approved for children as young as six months and for adults with confirmed WAS gene mutations, Waskyra is intended for patients eligible for hematopoietic stem cell transplantation (HSCT) but lacking a suitable HLA-matched related donor, according to an FDA release.

The therapy uses a patient’s own hematopoietic stem cells, genetically corrected ex vivo to add functional copies of the WAS gene. After reduced-intensity conditioning, the modified cells are infused to reconstitute normal immune and blood cell function, the agency says

WAS causes severe immunodeficiency, bleeding problems, eczema, and heightened risks of autoimmunity and malignancies, according to the release. Allogeneic HSCT is currently the only disease-modifying treatment, but is limited by donor availability and offers the greatest benefit when performed early in life.

Approval was based on data from two open-label, single-arm clinical studies and an expanded access program totaling 27 patients with severe disease. According to FDA, the data show durable clinical benefit, including a 93% reduction in severe infections in the six- to 18-month period after treatment compared with the 12 months prior. Moderate and severe bleeding events were reduced by 60% in the first post-treatment year, and most patients reported no moderate or severe bleeding four years after infusion, it says.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome