All articles tagged with #amt 130
UniQure’s Huntington’s disease gene therapy AMT-130 is back on track for FDA accelerated approval after the ouster of Vinay Prasad and other Trump-era regulators; the agency now accepts a standard-care control arm instead of sham surgeries, with a filing anticipated in Q3.
The FDA rejected uniQure's plan to file AMT-130 using phase 1/2 data with an external control and demanded a prospective, randomized, sham-controlled phase 3 trial, dampening near-term approval hopes and triggering a strategic review by the company; uniQure will discuss trial designs with the FDA and add a four-year durability analysis to the phase 1/2 data.
A small trial of uniQure’s AMT-130 gene therapy in 29 Huntington’s disease patients showed reduced production of the mutant huntingtin protein, slower cognitive decline, and lower levels of neurofilament light in cerebrospinal fluid over three years, suggesting a potential disease-modifying effect. Results were reported in a press release and have not yet undergone peer review, but they point to a possible treatment that could be used earlier in the disease course, while researchers continue to identify early biomarkers and expand the treatment window before motor symptoms appear.
uniQure announced that the FDA no longer considers Phase I/II data versus an external control sufficient as primary evidence for a BLA submission for its Huntington's disease gene therapy AMT-130, creating uncertainty about the approval timeline. The company plans urgent discussions with the FDA and continues regulatory engagement in the EU and UK, despite holding breakthrough therapy and RMAT designations.
A groundbreaking gene therapy called AMT-130 has shown promising results in slowing the progression of Huntington's disease in a clinical trial, with treated patients experiencing 75% less disease progression and lower neuronal damage markers, potentially offering a new hope for this currently incurable condition.
A small, early study suggests that the gene therapy AMT-130 may slow Huntington's disease progression, but results are preliminary, unreviewed, and require further research to confirm efficacy and safety.
Researchers in the UK have trialed a promising gene therapy, AMT-130, which can slow Huntington's disease progression by up to 75% over three years, potentially offering the first effective treatment for this fatal neurodegenerative disorder.
UniQure's experimental gene therapy, AMT-130, showed promising results in slowing Huntington's disease progression by 75%, leading to a over 240% surge in its stock price and plans to seek FDA approval by 2026, offering hope for a disease-modifying treatment.
An experimental gene therapy called AMT-130 from Uniqure significantly slowed Huntington's disease progression by 75% over three years, potentially paving the way for the first genetic treatment approval for this neurodegenerative disorder.