The FDA has pulled a U-turn on uniQure’s Huntington’s disease gene therapy, giving the biotech the OK to file for accelerated approval after previously demanding data from another trial.
After suffering the setback in March, uniQure arranged another meeting with the FDA to discuss the next steps. At the meeting, the FDA said a three-year analysis from a phase 1/2 study would be acceptable as the primary basis of a filing for accelerated approval of AMT-130 in Huntington’s, uniQure said in a June 17 release.
The FDA previously ruled the three-year analysis insufficient to support a filing for accelerated approval.
With the FDA changing its tune, uniQure plans to file for accelerated approval in the third quarter of this year. The biotech’s share price surged 80% to $48.51 in premarket trading on the back of the news. Investors also drove small upticks in the share price of biotechs including Rezolute and Regenxbio, potentially reflecting hopes that the U-turn indicates the FDA is increasingly flexible on rare disease evidence requirements.
The FDA’s reversal of its opposition to uniQure’s filing could indicate the agency’s “appreciation of the challenges in rare disease following the overhaul of its leadership,” Guggenheim Securities analysts said in a note to investors. Former FDA leaders Marty Makary, M.D., and Vinay Prasad, M.D., held top positions at the agency when uniQure first discussed filing for accelerated approval, but both have since left the agency.
UniQure still needs to reach agreement with the FDA on the path to full approval. The agency “seeks to align on the confirmatory study design” before the submission for accelerated approval, uniQure said. UniQure and the FDA are seeking alignment on using a concurrent control for standard-of-care therapy instead of the previously requested sham procedure.
The company said it is “committed to conducting the confirmatory study without delay.” Confirmatory trials typically need to be underway before the FDA grants accelerated approval. In some cases, the FDA may require the sponsor to have fully enrolled the confirmatory trial at the time of approval.
