UniQure, maker of HD drugs, seeks FDA approval for gene therapy
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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 supporting the application.
UniQure said the FDA said in a recent meeting that a three-year analysis of a Phase 1/2 trial would support accelerated approval of UniQure’s gene therapy for Huntington’s disease, a rare inherited disease that progressively 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 to submit a marketing application and accelerate approval of the therapy based on existing clinical data. The FDA “remains committed to working with UniQure to find a regulatory path that benefits patients with Huntington’s disease and their families while maintaining the agency’s commitment to gold standard science,” the official said in a statement.
UniQure shares rose 70% on Wednesday.
The FDA’s new guidance represents a stunning about-face from March, when the regulator told Uniqure that its clinical trial data did not support an application and publicly criticized the company. UniQure became a prime example in a series of setbacks in which companies said the FDA changed its previous guidance, hitting rare disease drugmakers particularly hard. Many of these decisions were made under former FDA Commissioner Marty Makary, who left the agency in May.
In an interview with CNBC’s Becky Quick in February, then-commissioner Makary described UniQure’s treatment without naming it, saying the agency was pressured to approve it even though it had shown “no benefit.” Then UniQure said the FDA could not agree 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 confirmed to reporters at the time that the FDA wanted UniQure to conduct a placebo-controlled trial to prove that its therapy “actually helps people.” Gene therapy is delivered directly into the brain through an hour-long operation, and UniQure has said it would be unethical to subject people to a sham procedure.
Huntington’s disease, also known as Huntington’s disease, is a neurodegenerative disease caused by a mutation in the huntingtin gene (HTT).
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Instead, the company used an external database to compare the course of the people treated with the typical course of Huntington’s disease. Using this approach, UniQure’s gene therapy slowed disease progression by 75% in a Phase 1/2 trial.
With the FDA’s approval, UniQure now plans to use the same data that was reviewed to support its application. Accelerated approval would allow UniQure’s treatment to be brought to market contingent on the company demonstrating benefit in another study.
UniQure said Wednesday that the FDA wants to guide 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 expects to finalize these plans before submitting the application.
UniQure is not the only company whose fortunes are changing following the departure of Makary and other senior executives, including former Center for Biologics Evaluation and Research director Vinay Prasad and former Center for Drug Evaluation and Research director Tracy Beth Høeg. Replenishment The company recently announced that it would apply for approval of its experimental melanoma drug for the third time.
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