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UniQure plans to seek FDA approval for its investigational gene therapy for Huntington’s disease, the company announced Wednesday, months after previous agency leaders criticized the evidence supporting the application.
UniQure said the FDA, at a recent meeting, announced that 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 disorder that gradually destroys nerve cells in the brain. Following this 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 process for submitting a marketing application and expedited approval of the treatment 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 upholding the agency’s commitment to gold-standard science,” the official said in a statement.
UniQure shares soared 70% on Wednesday.
The FDA’s new guidance represents a stunning reversal from March, when the regulator told Uniqure that its clinical trial data would not support an application and publicly criticized the company. UniQure became a prime example of a series of reversals in which companies said the FDA changed its previous guidance, hitting makers of drugs for rare diseases particularly hard. Many of these decisions were made under the leadership 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 the UniQure treatment without naming it, saying the agency was pressured to approve it even though it had “no benefit.” Next, UniQure said the FDA could not accept that data from a clinical trial comparing UniQure’s gene therapy to an external 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 prove that its therapy “actually helps people.” Gene therapy is delivered directly to the brain through a surgery that lasts hours, and UniQure has said it would be unethical to force people to undergo a sham procedure.
Huntington’s disease, also known as Huntington’s chorea, is a neurodegenerative disease caused by a mutation in the huntingtin, HTT, gene.
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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 this 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 has come under scrutiny to support its application. Accelerated approval would allow UniQure’s treatment to go to market provided the company proves its benefits in another study.
UniQure said Wednesday that the FDA wants to align with the design of this study, including comparing the treatment to the current standard of care rather than a sham procedure. UniQure said it is committed to conducting this study and hopes to finalize these plans before submitting its application.
UniQure is not the only company to see its fortunes reverse after the departure of Makary and other top executives, 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 will seek approval for its experimental melanoma drug for the third time.