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UniQure plans to seek FDA approval for its experimental gene therapy for Huntington’s disease, the company said Wednesday, months after former agency leaders criticized the evidence supporting the application.
UniQure said the FDA at a recent meeting revealed 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 genetic disease that slowly 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.
UniQure shares surged 70% on Wednesday.
The FDA’s new guidance represents a dramatic reversal from March, when the regulator told Uniqure that its clinical trial data would not support the application and publicly criticized the company. UniQure has been a prime example of a series of setbacks where companies say the FDA has changed its previous guidance, hitting rare drug makers especially hard. Many of those decisions took place under former FDA Commissioner Marty Makary, who left the agency in May.
In a February interview with CNBC’s Becky Quick, former Commissioner Makary explained UniQure’s treatment without naming it, saying the agency was pressured to approve it even though it showed no “profit.” Then UniQure said the FDA could not agree that data from a clinical trial comparing UniQure’s gene therapy with an external control was sufficient to support the application.
A top FDA official at the time confirmed to reporters that the FDA wanted UniQure to conduct a placebo-controlled study to prove that its treatment “really works for people.” Gene therapy is delivered directly to the brain in a surgery that takes hours, and UniQure said it would not be wrong to fake the procedure.
Instead, the company compared the progress of people receiving the treatment to the normal progression of Huntington’s disease using an external database. Using that approach, UniQure’s gene therapy reduced disease progression by 75% in a Phase 1/2 study.
Huntington’s disease, also known as Huntington’s chorea, is a neurodegenerative disease caused by mutations in the huntingtin gene, HTT.
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With the FDA’s blessing, UniQure now plans to use the same data it previously analyzed to support its use. The accelerated approval would allow UniQure’s treatment to come to market on the condition that the company proves benefit in further research.
UniQure on Wednesday said the FDA wants to comply with the design of that study, which includes comparing the treatment to the current standard of care rather than a sham procedure. UniQure said it is committed to doing that research and expects to complete those plans before submitting its bid.
UniQure isn’t the only company that has seen its fortunes decline after the departure of Makary and other top leaders, including former Center for Biological Evaluation and Research director Vinay Prasad and former Center for Drug Evaluation and Research director Tracy Beth Høeg. Answer recently announced that it will seek approval for its investigational melanoma drug for a third time.
