Nov 2 (Reuters) – Dutch gene therapy developer uniQure said on Monday the US FDA had accepted its application for its Huntington’s disease treatment and would decide on the drug by May 1, 2027.
US-listed shares of the company were up 2.5% in premarket trading.
Here are some details:
• The development comes months after uniQure said that AMT-130 failed to slow disease progression in a four-year trial, sending its shares sharply lower
• The FDA’s Monday decision to review AMT-130 ends a lengthy standoff with uniQure, during which a senior agency official described the treatment as a “failed product”
• The regulator later reversed course and agreed to review the application
• The FDA granted the therapy a faster review, uniQure said. The agency is also considering holding a meeting of outside experts to discuss the application
• UniQure’s application is based on early- to mid-stage study results comparing treated patients with a group of patients from an external Huntington’s disease database. The company said it has treated 51 patients across its studies so far
• The FDA is reviewing the application under its accelerated approval pathway, which can allow speedier approval for treatments targeting serious diseases based on early evidence, while requiring the company to confirm the benefit in future studies
• UniQure plans to begin US patient screening by year-end for a 200-patient follow-up trial, with two-thirds receiving the gene therapy and the rest standard care
(Reporting by Padmanabhan Ananthan in Bengaluru; Editing by Shinjini Ganguli)
