FDA Clears UniQure to Submit Huntington's Disease Treatment for Approval
The U.S. Food and Drug Administration (FDA) has reversed its earlier opposition, allowing biotech company UniQure to proceed with filing for U.S. approval of its experimental Huntington's disease treatment, AMT-130. UniQure plans to submit a marketing application for accelerated approval in the third quarter of this year. This decision follows a recent meeting where the FDA accepted a three-year analysis from an early-stage study demonstrating patient benefit as sufficient for supporting the application.
The U.S. Food and Drug Administration has reversed its previous opposition to an experimental treatment for Huntington's disease, clearing the way for biotech company UniQure to submit its drug for U.S. approval.
UniQure announced its intention to file a marketing application for accelerated approval of the treatment, known as AMT-130, during the third quarter. This development follows a recent meeting with FDA officials, where the agency deemed a three-year analysis from an early-stage study acceptable to support a marketing application. The analysis reportedly showed a benefit for patients with Huntington's disease.
This marks a change in stance, as former FDA officials, including Commissioner Marty Makary and Vinay Prasad, the agency's top regulator for cell and gene therapies, had previously concluded that the same AMT-130 data were insufficient to support a marketing application.
According to STAT News, UniQure is now poised to advance its regulatory process for this closely watched treatment.
