UniQure, the Netherlands-based biotechnology company, intends to formally ask the Food and Drug Administration to approve its for Huntington’s disease gene therapy now that the two parties are more aligned on the closely watched treatment.
UniQure said Wednesday that, during a recent meeting, FDA staff agreed three years of data gathered from a key trial of the therapy would be enough to support an approval application. As such, the company expects to file one sometime between July and the end of September. The FDA has requested another trial be conducted to confirm the treatment’s effects and, according to the UniQure, the agency wants to make sure both sides see eye to eye on this study’s design before a marketing application gets submitted.
The update is a major about-face for the FDA and a reflection of recent changes in its leadership. Marty Makary resigned as commissioner in mid-May, just days before the firing of Tracy Beth Høeg, who served as acting director of the agency’s main drug review division. Those shake-ups came a little over two months after Vinay Prasad, who oversaw the office that regulates vaccines and gene therapies, departed the FDA for a second time.
Prasad played a particularly important role in what would became a winding, highly publicized saga for UniQure’s “AMT-130” therapy. An oncologist by training, Prasad had been a staunch critic of the ways in which the FDA had tried to expedite the review of certain genetic medicines. Some speculated that, as UniQure received unexpected pushback from the agency, Prasad was either directly or tangentially involved.
