FDA Policy/General

Sangamo Begins Rolling BLA for Fabry Gene Therapy

Sangamo Therapeutics has begun a rolling BLA submission seeking accelerated approval of its experimental Fabry disease gene therapy, following results from a registrational study that the agency has agreed can serve as the primary basis for approval. The company says that data from its Phase 1/2 STAAR study support the potential approval of isaralgagene civaparvovec (ST-920), a one-time adeno-associated virus gene therapy designed to address the underlying cause of Fabry disease. FDA has agreed that improvement in kidney function, measured by mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks, can be used as an intermediate clinical endpoint under the accelerated approval pathway, it adds.

Sangamo says that a 4/2025 STAAR study data cutoff showed a positive mean annualized eGFR slope at 52 weeks across all 32 dosed patients, indicating improved renal function in a disease typically marked by progressive kidney decline. It says that all 32 treated patients have enrolled into a long-term follow-up study.

Beyond kidney outcomes, the company reports stable cardiac structure and biomarkers over one year, along with sustained expression of alpha-galactosidase A activity for up to 4.5 years in the longest-treated patient. Statistically significant improvements in quality-of-life measures were also observed.

Fabry disease is a rare, inherited lysosomal storage disorder that leads to progressive damage of the kidneys, heart, nervous system and other organs, often resulting in reduced life expectancy.

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