UniQure plans to file Huntington’s disease gene therapy with FDA after leadership shift eases data requirements

UniQure, a Dutch biotech, announced it will seek FDA approval for its gene therapy targeting Huntington’s disease later this summer.

The company said FDA staff indicated that three years of data from a pivotal trial are sufficient to support an application, and it expects to file between July and September.

The agency, however, has asked for an additional trial to confirm the therapy’s effect and wants agreement on the study design before a marketing submission.

Analysts view the move as a notable regulatory shift following recent leadership changes at the FDA, including the resignation of commissioner Marty Makary and the departure of acting director Tracy Beth Høeg.