Biologics

FDA Shifts Stance on AMT-130 BLA Data

Gene therapy company uniQure says that FDA indicated during a recent pre-BLA meeting that the agency may no longer view the company’s Phase 1-2 data for its Huntington’s disease (HD) therapy AMT-130, analyzed against an external control, as sufficient to serve as primary evidence to support a planned submission.

The company says that while final meeting minutes have not yet been received, the feedback marks a “key shift” from prior communications over the past year, during which the agency had suggested the current data could form the basis for an accelerated approval filing. As a result, the timeline for a BLA submission is now unclear, it says.

In September, the company reported favorable data from the study. The study met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a statistically significant (75%) slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale at 36 months compared to a propensity score-matched external control, according to the company.

“We are surprised by the FDA’s feedback at the recent pre-BLA meeting, which is a drastic change from the guidance provided in November 2024,” CEO Matt Kapusta is quoted in a release as saying. “We remain fully committed to working with the FDA to determine the best path forward.”

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