Biologics

uniQure Files BLA for Huntington’s Gene Therapy

uniQure has submitted a BLA seeking accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for treating Huntington’s disease. The company’s submission requested a priority review.

The filing is based on a three-year data analysis from a Phase I/II clinical study of ifezuntirgene inilparvovec, compared to a propensity score-matched external control derived from the Enroll-HD natural history database. The company expects to present a four-year data analysis from clinical studies before the end of the current quarter.

Earlier this year, UniQure was tangled in a dispute with the agency over then-CBER director Vinay Prasad’s insistence on a new randomized trial, saying such designs are necessary in diseases like Huntington’s where outcomes can vary widely and subjective endpoints may be influenced by expectations (see earlier story).

After Prasad’s departure, FDA reversed course and told UniQure that data from its ongoing Phase I/II study could serve as the primary basis for a BLA. FDA also requested alignment on the design of a post-approval confirmatory study before the filing is submitted.

AMT-130 is described as an investigational one-time gene therapy designed to lower production of the mutant huntingtin protein that causes Huntington's disease, a rare inherited neurodegenerative disorder characterized by progressive motor, cognitive, and psychiatric decline. No therapies are currently approved to slow disease progression.

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