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UniQure plans to seek FDA approval of its experimental gene therapy for Huntington’s disease, the company said Wednesday, months after previous agency leaders criticized the evidence backing the application.
UniQure said the FDA in a recent meeting communicated that a three-year analysis from a Phase 1/2 study would support an accelerated approval of UniQure’s gene therapy for Huntington’s, a rare hereditary 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 have agreed on a path for submission for a marketing application and accelerated approval of the therapy based on the existing clinical data. The FDA “remains committed to working with UniQure to identify a regulatory pathway that serves patients with Huntington’s disease and their families, while upholding the agency’s commitment to gold-standard science,” the official said in a statement.
Shares of UniQure soared 70% on Wednesday.
The new FDA guidance represents a stunning reversal from March, when the regulator told Uniqure that its clinical trial data wouldn’t support an application and publicly criticized the company. UniQure became a prime example in a series of reversals where companies said the FDA had changed its previous guidance, hitting rare disease drugmakers especially hard. Many of those decisions happened under 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.” Then UniQure said the FDA couldn’t agree that data from a clinical trial comparing UniQure’s gene therapy to an external control are sufficient to support an application.
A senior FDA official at the time confirmed to reporters that the FDA wanted UniQure to run a placebo-controlled trial to prove its therapy “actually helps people.” The gene therapy is administered directly into the brain through an hours-long surgery, and UniQure has said it would be unethical to make people undergo a sham procedure.
Huntington’s disease, also known as Huntington’s chorea, is a neurodegenerative disease due to a mutation in the huntingtin gene, HTT.
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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 outside 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 prove the benefit in another study.
UniQure on Wednesday said the FDA wants to align on that study’s design, including comparing the treatment to the current standard of care rather than a sham procedure. UniQure said it’s committed to conducting that study and expects to finalize those plans before submitting its application.
UniQure isn’t 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 it would seek approval of its experimental melanoma drug for a third time.
