Drugs

uniQure Reports Favorable Data in Huntington’s Trial

uniQure says its investigational gene therapy AMT-130 significantly slowed disease progression in Huntington’s disease patients, which the company calls the most convincing evidence to date of a disease-modifying effect in the fatal neurodegenerative disorder. In a pivotal Phase 1-2 study, patients who received a high dose of AMT-130 experienced a 75% slowing of disease progression over three years, measured by the composite Unified Huntington’s Disease Rating Scale, compared with matched external controls. The therapy also met a key secondary endpoint, showing a 60% slowing of decline in “total functional capacity.”

The study followed 29 patients treated with either high or low doses of AMT-130, with outcomes compared against more than 1,500 patients in the Enroll-HD natural history dataset. At the 36-month mark, those given the high dose not only showed slowing of motor and cognitive decline on multiple measures, but also a mean reduction of cerebrospinal neurofilament light protein, a biomarker of neurodegeneration, the company says.

uniQure plans to meet with FDA later this year ahead of a BLA submission in the first quarter of 2026. AMT-130 already holds a breakthrough therapy and regenerative medicine designations.

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

Read more

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