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unicure The company plans to seek FDA approval for its experimental gene therapy for Huntington’s disease, the company announced Wednesday, months after the former commissioner criticized the evidence supporting its application.
UniQure said the FDA informed it at a recent meeting that a three-year analysis from a Phase 1/2 trial supports early approval of UniQure’s gene therapy for Huntington’s disease, a rare genetic disease that gradually destroys nerve cells in the brain. As a result of the meeting, UniQure plans to file an application with the FDA in the third quarter of this year.
FDA officials confirmed that the agency and the company have agreed on a path to accelerate marketing application submissions and treatment approvals based on existing clinical data. The FDA “remains committed to working with UniQure to identify regulatory pathways that benefit Huntington’s disease patients and their families, while maintaining FDA’s commitment to the highest standards of science,” officials said in a statement.
UniQure’s stock price soared 70% on Wednesday.
The FDA’s new guidance marks a stunning reversal from March, when the regulator publicly criticized UniQure, telling it its clinical trial data did not support its application. UniQure became a key example in a series of reversals in which companies claim the FDA changed previous guidance, hitting rare disease drug companies particularly hard. Many of these decisions were made under former FDA Commissioner Marty McCulley, who left the agency in May.
In a February interview with CNBC’s Becky Quick, then-Secretary McCurry described UniQure’s treatment without naming names, saying he was under pressure to approve UniQure even though it had been shown to be “unprofitable.” UniQure then stated that the FDA could not agree that the data from clinical trials comparing UniQure’s gene therapy with an external control were sufficient to support the application.
A senior FDA official at the time acknowledged to reporters that the agency wanted to conduct a placebo-controlled trial to prove that UniQure’s treatment “actually helps people.” Gene therapy is administered directly to the brain through a surgery that lasts several hours, but UniQure said it would be unethical to subject people to a fake surgery.
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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Instead, the company used an external database to compare the progress of people treated with the typical progression of Huntington’s disease. UniQure’s gene therapy using this approach slowed disease progression by 75% in a Phase 1/2 trial.
With FDA permission, UniQure now plans to use the same data that came under scrutiny to support its applications. Early approval would allow UniQure’s treatment to be brought to market, provided the company proves benefit in separate studies.
UniQure said Wednesday that the FDA wants to align its study plan with comparing its treatment to current standard treatments rather than sham treatments. UniQure said it is committed to carrying out its research and plans to finalize plans before submitting the application.
UniQure is not the only company whose fortunes have reversed following the resignations of senior executives such as McCurry, Vinay Prasad, former director of the Center for Biologics Evaluation and Research, and Tracy Beth Hogue, former director of the Center for Pharmaceutical Evaluation and Research. Replimune recently announced that it will seek approval for a third time for its experimental melanoma drug.
