FDA’s rebuff of uniQure raises uncertainty over external control standards

The latest about-face by FDA on a planned regulatory submission has resulted in gene therapy developer uniQure losing half its value on Monday, and the biotech is mulling next steps as it considers the future of its Huntington’s disease program.

uniQure N.V. (NASDAQ:QURE) said early Monday that FDA is no longer open to a BLA submission seeking accelerated approval of AMT-130 using external matched controls. The company said in September that the HTT-targeted gene therapy had shown disease-modifying effects in a Phase I/II study to treat Huntington’s disease, lifting shares and leading to a $300 million offering...