The U.S. Food and Drug Administration has determined that the Phase I/II data for uniQure’s investigational gene therapy AMT‑130, intended to treat Huntington’s disease, is insufficient to support a Biologics License Application. The decision follows the agency’s review of the study’s comparison to a propensity‑score matched external control derived from the Enroll‑HD natural history dataset.
The agency’s feedback represents a material shift from guidance issued in November 2024 and a change from the Type B meeting guidance that uniQure had previously received. The FDA now requires additional evidence to demonstrate safety and efficacy before a marketing application can be considered.
UniQure’s senior leadership stated that the company will explore alternative study designs, including a prospective randomized controlled trial, and will seek additional data on long‑term safety and biomarker outcomes. The company also indicated that it will engage with the FDA to clarify the data requirements and to outline a revised development plan.
AMT‑130 has been the company’s flagship product and a key driver of future revenue. The therapy targets a rare, fatal neurodegenerative disorder for which no disease‑modifying treatments are currently approved. The setback introduces uncertainty into the projected timeline for regulatory approval and commercialization, potentially affecting the company’s growth prospects.
The Huntington’s disease treatment landscape includes other gene‑therapy candidates, such as Wave Life Sciences’ allele‑specific silencing therapy and Prilenia Therapeutics’ pridopidine, which is under review in Europe. UniQure’s position as a leading candidate is now challenged by the need for additional data and the evolving regulatory expectations.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.