Biologics

Sangamo to Seek Accelerated OK on Gene Therapy

Sangamo Therapeutics says it reached agreement with FDA on a “clear regulatory pathway to accelerated approval for the company’s gene therapy candidate ST-920 (isaralgagene civaparvovec) for treating Fabry disease. The FDA has agreed in a Type B interaction that data from the ongoing Phase 1/2 STAAR study can serve as the primary basis for approval under the accelerated approval program, using eGFR [estimated glomerular filtration rate] slope at 52 weeks across all patients as an intermediate clinical endpoint,” it says.

The complete dataset to support accelerated approval is said to become available in the first half of 2025, and a potential BLA submission could be filed in the second half of 2025, three years ahead of previous estimates because it avoids the requirement for an additional registrational study to establish clinical efficacy, according to Sangamo.

The company says an analysis of clinical data from the Phase 1/2 STAAR study showed “encouraging safety and efficacy data, including promising preliminary evidence of improved kidney function. Renal manifestations, such as proteinuria or a decreased glomerular filtration rate, occur early in life in almost all male, and in many female, patients with Fabry disease, and can lead to end-stage renal disease and early death. In the 18 male and female patients treated with isaralgagene civaparvovec with more than one year of follow-up data, statistically significant improvements were observed in both mean and median eGFR levels, resulting in a positive annualized eGFR slope.”

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