UniQure Shares Surge 70% After FDA Reversal on Huntington's Gene Therapy

UniQure plans to seek FDA approval of its experimental gene therapy for Huntington's disease, the company said Wednesday. The announcement came months after former agency leaders publicly criticized the evidence behind the treatment.

Shares of UniQure (QURE) jumped 70% on the news.

The FDA in a recent meeting told UniQure that a three-year analysis from a Phase 1/2 study would support an accelerated approval application, the company said. UniQure now expects to submit that application in the third quarter of this year.

The new guidance marks a sharp reversal from March, when the regulator told UniQure its clinical trial data would not support an application. Former FDA Commissioner Marty Makary, who left the agency in May, had described the treatment in a February CNBC interview, saying the agency was pressured to approve it even though it showed "no benefit." A senior FDA official at the time said the agency wanted a placebo-controlled trial to prove the therapy "actually helps people."

UniQure's gene therapy is administered directly into the brain through an hours-long surgery. The company argued it would be unethical to make patients undergo a sham procedure. Instead, it compared the progression of treated patients to the typical course of Huntington's disease using an external database. That approach showed a 75% slowing of disease progression in the 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 bring the treatment to market on the condition that the company prove the benefit in a later study. UniQure 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. The company said it expects to finalize those plans before submitting its application.

UniQure is not the only company to see its fortunes reverse after Makary and other senior FDA leaders departed. Replimune recently announced it would seek approval of its experimental melanoma drug for a third time.