India, June 17 -- uniQure N.V. (QURE) said the feedback came during a recent Type B meeting with the agency, where regulators confirmed that the three-year analysis from its Phase I/II study would be acceptable as the primary foundation for a BLA submission for AMT-130, its gene therapy for Huntington's disease. The company now plans to submit the application in the third quarter of 2026. A closer look at the FDA guidance
The FDA also asked to finalize the design of the required confirmatory study before the BLA is filed, including the potential use of concurrent controls on standard-of-care therapy instead of a sham procedure-a notable shift in trial expectations for this rare neurodegenerative disorder. Regulators emphasized they would w...