The FDA granted a priority review to uniQure (NASDAQ: QURE) for its Biologics License Application (BLA) for ifezuntirgeneinilparvovec, a gene therapy for Huntington’s disease. The BLA was submitted under the accelerated approval pathway. Matt Kapusta, chief executive officer of uniQure, said the acceptance marks an important milestone for the Huntington’s disease community, which has waited generations for a treatment that changes the course of this devastating disease.
He added that the company, backed by compelling data from our Phase I/II studies,
believes ifezuntirgeneinilparvovec has the potential to become the first disease modifying treatment for Huntington’s disease.
Sevasemten Fails in Becker Muscular Dystrophy Trial
In a separate development, Servier’s investigational agent sevasemten failed to meet its primary or secondary endpoints in the GRAND CANYON trial for Becker muscular dystrophy (BMD). A recent news release from Servier indicates the outcome contradicts a long history of encouraging data for a drug that was on track to be the first approved treatment for the condition, for which no options currently exist.
GRAND CANYON is the main cohort of a broader clinical program (NCT05291091) that also includes the phase 2 CANYON study. The trial assigned approximately 120 adults with BMD 2:1 to receive oral sevasemten or placebo over an 18-month period, using change in the North Star Ambulatory Assessment (NSAA) as the main measure of success. Servier reported that sevasemten did not show a statistically significant effect on the NSAA and that various secondary endpoints were also not achieved.
We are thrilled to announce that the FDA has accepted for review the BLA for ifezuntirgene inilparvovec (AMT-130), an investigational #genetherapy for #HuntingtonsDisease. The application has been granted Priority Review with a PDUFA date of May 1, 2027. https://t.co/DQMYULhuR4 pic.twitter.com/BM8Wjv1ale
1-Year KYSA-8 Findings Show Sustained Improvements
One-year data from the phase 2 KYSA-8 study, which looked at a single infusion of miv-cel in adults with SPS, were shared at the 10th Joint ACTRIMS-ECTRIMS Meeting (MSToronto 2026), taking place October 21-23, 2026, in Toronto, Ontario, Canada. Researchers noted continued progress in assessments of walking ability, neurologic disability, and stiffness. The therapy also exhibited a positive safety profile, with no instances of high-grade cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS).
More on Huntington
$QURE Q3 2026 earnings: FDA fast-tracks uniQure's Huntington's drug, but four-year data weakens uniQure, a developer of one-time gene therapies, cleared its main regulatory hurdles for Huntington's disease while posting mixed four-year trial results. The US regulator accepted… pic.twitter.com/e0HESYD2zu
