Huntington’s disease drug maker UniQure is seeking US Food and Drug Administration (FDA) approval for its gene therapy

UniQure The company said Wednesday it plans to seek FDA approval for its experimental gene therapy for Huntington’s disease, months after the agency’s former leaders criticized the evidence supporting the application.

UniQure said the US Food and Drug Administration announced at its recent meeting 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 genetic disease that gradually destroys nerve cells in the brain. As a result of 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 have agreed on a path to submit a marketing application and accelerate approval of the treatment based on 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 maintaining the agency’s commitment to scientific gold standards,” the official said in a statement.

UniQure shares rose 70% on Wednesday.

The new FDA guidance represents a stunning reversal from in March, when the regulator told Uniqure that its clinical trial data would not support the app and publicly criticized the company. UniQure has become a prime example in a series of reversals as companies say the Food and Drug Administration has changed its previous guidance, severely hurting rare disease drug makers. Many of these decisions were made under former FDA Commissioner Marty McCurry, who left the agency in May.

In a February interview with CNBC’s Becky Quick, then-Commissioner McCurry described the UniQure transaction without naming it, saying the agency was pressured to approve it even though it showed “no benefit.” UniQure then said the FDA was unable to agree that data from a clinical trial comparing UniQure’s gene therapy with an outside control were 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 treatment “actually helps people.” The gene therapy is administered directly to the brain through an hours-long surgical procedure, and UniQure said it would be unethical to subject people to sham procedures.

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 audited data to support its application. Rapid approval would allow UniQure’s treatment to reach the market provided the company proves the benefit in another study.

UniQure said Wednesday that the FDA wants to align with the design of that study, including comparing the treatment to current standards of care rather than a sham procedure. UniQure said it is committed to conducting that study and expects to finalize those plans before submitting its application.

UniQure is not the only company to see its fortunes decline following the departure of McCary and other senior leaders, including former Center for Biological Evaluation and Research Director Vinay Prasad and former Center for Drug Evaluation and Research Director Tracey Beth Hoge. to reply It recently announced that it would seek approval for an experimental skin cancer drug for a third time.