UniQure to seek FDA approval for Huntington’s disease gene therapy after previous clash with agency

UniQure plans to seek FDA approval of its experimental gene therapy for Huntington’s disease, the corporate stated Wednesday, months after previous agency leaders criticized the proof backing the appliance.

UniQure stated the FDA in a current assembly communicated {that a} three-year evaluation from a Section 1/2 examine would assist an accelerated approval of UniQure’s gene therapy for Huntington’s, a uncommon hereditary disease that regularly destroys nerve cells within the mind. Because of the assembly, UniQure plans to submit its utility to the FDA within the third quarter of this yr.

An FDA official confirmed that the agency and the corporate have agreed on a path for submission for a advertising utility and accelerated approval of the therapy primarily based on the present medical knowledge. The FDA “stays dedicated to working with UniQure to determine a regulatory pathway that serves sufferers with Huntington’s disease and their households, whereas upholding the agency’s dedication to gold-standard science,” the official stated in a press release.

Shares of UniQure soared 70% on Wednesday.

The brand new FDA steerage represents a shocking reversal from March, when the regulator advised Uniqure that its medical trial knowledge would not assist an utility and publicly criticized the corporate. UniQure turned a major instance in a collection of reversals the place firms stated the FDA had modified its previous steerage, hitting uncommon disease drugmakers particularly onerous. Lots of these selections occurred underneath former FDA Commissioner Marty Makary, who left the agency in Might.

In a February interview with CNBC’s Becky Fast, then-Commissioner Makary described UniQure’s remedy with out naming it, saying the agency was pressured to approve it despite the fact that it confirmed “no profit.” Then UniQure stated the FDA could not agree that knowledge from a medical trial evaluating UniQure’s gene therapy to an exterior management are ample to assist an utility.

A senior FDA official on the time confirmed to reporters that the FDA wished UniQure to run a placebo-controlled trial to show its therapy “truly helps individuals.” The gene therapy is run instantly into the mind by means of an hours-long surgical procedure, and UniQure has stated it might be unethical to make individuals bear a sham process.

As a substitute, the corporate in contrast the development of people that obtained the remedy to the everyday development of Huntington’s disease utilizing an outdoor database. Utilizing that method, UniQure’s gene therapy slowed disease development by 75% in a Section 1/2 trial.

With the FDA’s blessing, UniQure now plans to use the identical knowledge that got here underneath scrutiny to assist its utility. An accelerated approval would enable UniQure’s remedy to come to market on the situation that the corporate show the profit in one other examine.

UniQure on Wednesday stated the FDA needs to align on that examine’s design, together with evaluating the remedy to the present customary of care reasonably than a sham process. UniQure stated it is dedicated to conducting that examine and expects to finalize these plans earlier than submitting its utility.

UniQure is not the one firm to see its fortunes reverse following the departure of Makary and different senior leaders, together with former Heart for Biologics Analysis and Analysis director Vinay Prasad and former Heart for Drug Analysis and Analysis director Tracy Beth Høeg. Replimune not too long ago introduced it might seek approval of its experimental melanoma drug for a 3rd time.