Thomas Fuller | SOPA Images | Light rocket | fake images
UniQure plans to seek FDA approval for its experimental gene therapy for Huntington's disease, the company said Wednesday, months after previous agency leaders criticized the evidence supporting the application.
UniQure said the FDA at a recent meeting said a three-year analysis of a Phase 1/2 study would support accelerated approval of UniQure's gene therapy for Huntington's disease, a rare inherited disease that gradually destroys nerve cells in the brain. As a result of the meeting, UniQure plans to submit its application to the FDA in the third quarter of this year.
An FDA official confirmed that the agency and the company agreed on a path to submitting a marketing application and expedited approval of the therapy based on existing clinical data. The FDA “remains committed to working with UniQure to identify a regulatory pathway that serves Huntington's disease patients and their families, while maintaining the agency's commitment to leading science,” the official said in a statement.
UniQure shares soared 70% on Wednesday.
The new FDA guidance represents a striking change from March, when the regulator told Uniqure that data from its clinical trials would not support an application and publicly criticized the company. UniQure became a prime example of a series of setbacks in which companies said the FDA had changed its previous guidance, especially affecting makers of rare disease drugs. Many of those decisions were made during the tenure of former FDA Commissioner Marty Makary, who left the agency in May.
In a February interview with CNBC's Becky Quick, then-Commissioner Makary described UniQure's treatment without naming it, saying the agency was pressured to approve it even though it showed “no benefit.” UniQure then said the FDA could not accept that data from a clinical trial comparing UniQure's gene therapy to an outside control was sufficient to support an application.
A senior FDA official at the time confirmed to reporters that the FDA wanted UniQure to conduct a placebo-controlled trial to show that its therapy “really helps people.” The gene therapy is administered directly into the brain through surgery that lasts a few hours, and UniQure has said it would be unethical to subject people to a sham procedure.
Huntington's disease, also known as Huntington's chorea, is a neurodegenerative disease due to a mutation in the Huntington's gene, HTT.
Kateryna Kon/Scientific Photo Library | Scientific photography library | fake images
Instead, the company compared the progression of people who received the treatment to the typical progression of Huntington's disease using an external database. Using that approach, UniQure's gene therapy slowed disease progression by 75% in a Phase 1/2 trial.
With the FDA's blessing, UniQure now plans to use the same data that came under scrutiny to support its application. An accelerated approval would allow UniQure's treatment to come to market on the condition that the company demonstrates benefit in another study.
UniQure said Wednesday that the FDA wants to align with the design of that study, including comparing the treatment to the current standard of care rather than a sham procedure. UniQure said it is committed to conducting that study and hopes to finalize those plans before submitting its application.
UniQure is not the only company to see its fortunes reverse following the departure of Makary and other senior leaders, including former Center for Biologics Evaluation and Research director Vinay Prasad and former Center for Drug Evaluation and Research director Tracy Beth Høeg. Replimune recently announced that it would seek approval of its experimental melanoma drug for the third time.
