UniQure announced its intention to submit an application for FDA approval of its gene therapy for Huntington's disease, following a recent meeting with the agency that indicated a three-year analysis from a Phase 1/2 study could support an accelerated approval.
This is a notable shift from earlier this year when the FDA criticized the company's clinical trial data and suggested that a placebo-controlled trial was necessary to demonstrate the therapy's efficacy.
The gene therapy, which is administered directly into the brain, reportedly slowed disease progression by 75% in the trial, using an external database for comparison rather than a placebo group, which UniQure argued would be unethical.
The FDA's new guidance allows UniQure to use the previously scrutinized data for its application, with the condition that the company conducts further studies to confirm the treatment's benefits. Following this announcement, UniQure's shares surged by 70%, reflecting investor optimism about the potential approval and market entry of the therapy.
This development is part of a broader trend where several companies have experienced reversals in FDA guidance following leadership changes at the agency, highlighting the evolving landscape for drug approvals in the rare disease sector