Shares of UniQure doubled Tuesday morning after the Netherlands-based biotechnology company trumpeted that its experimental treatment for Huntington’s disease could be eligible for a speedy kind of approval.
Known as AMT-130, the treatment is one of UniQure’s most advanced and closely watched assets. It’s a gene therapy designed to enter brain cells and block the genetic instructions for the protein that causes Huntington’s. UniQure has been periodically providing updates from two clinical trials testing multiple doses of AMT-130, and in July said the therapy appears to be slowing the progression of Huntington’s in patients who are early on in their disease.
Several months after disclosing those results, UniQure met with the Food and Drug Administration to discuss the program’s next steps. The company is now saying that during this late November meeting, the two parties agreed on some of the key elements UniQure would need if it planned to ask for “accelerated approval” — a type of clearance which allows drugs with limited supporting data to enter the market while their developers collect more evidence to confirm they are effective.
According to UniQure, the FDA said data from the ongoing trials of AMT-130 may serve as the main basis for an accelerated approval application, so long as the data are compared to an external control group of people who have early Huntington’s disease and are similar to the study participants. As such, the company would not need to conduct an additional trial before submitting its application.
UniQure said the two parties also agreed on ways to measure the effectiveness of AMT-130.
According to the company, FDA staff think a well-known scale for evaluating the progression of Huntington’s could be the main tool that shows AMT-130 is reasonably likely to benefit patients. The staff also believe reductions in “neurofilament light chain,” a protein tied to nerve cell damage, could be used as supportive evidence. Notably, Biogen’s Qalsody, a drug for ALS, won FDA approval last year because of its effects on neurofilament, which then encouraged other brain drug developers to prioritize measuring the protein in their studies.
Meetings like the one between UniQure and the FDA aren’t public, meaning the agency’s position on AMT-130 isn’t entirely clear. But the FDA’s stance, as described by UniQure, does seem fitting, given recent views expressed by top-ranking officials.
In May, Peter Marks, head of the FDA office that reviews gene therapies, said the agency changed its mindset over the past several years and is now more aggressively looking for ways to speed the development and approval of gene therapies for rare diseases.
That’s good news for companies like UniQure, which want to recoup their research investments sooner through accelerated approvals. Just last month, Maryland-based gene therapy developer Regenxbio said it had reached an agreement with the FDA about a path to accelerated approval for a Duchenne muscular dystrophy treatment. The company’s shares subsequently climbed about 11%.
UniQure’s stock bump was far greater, with shares up more than 100% by noon Tuesday.
In a note to clients, Stifel analyst Paul Matteis wrote that while there are still outstanding questions, alignment with the FDA on an accelerated approval pathway is a “best-case scenario” and a “big win” for UniQure, especially since investors had low expectations that such an option would materialize.
UniQure said it has begun “readiness activities” for its approval application and intends to further engage with the FDA in the first half of 2025.
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- Source: https://www.biopharmadive.com/news/uniqure-huntington-fda-accelerated-approval-pathway-gene-therapy/735123/