In a major setback, Uniqure said Monday that the timing of when it can file its experimental and promising Huntington’s disease treatment for approval with the Food and Drug Administration “is now unclear,” raising the prospect that the biotech may need more data.
In a statement, Uniqure said that at a recent meeting with the FDA about the treatment, a gene therapy known as AMT-130, the agency signaled that it “no longer agrees” that existing data from a Phase 1/2 study with an external control group are adequate for an approval submission. The company called it “a key shift from prior communications with the FDA” in multiple meetings over the past year.
Uniqure shares were down some 60% in pre-market trading following the company’s announcement.
STAT+ Exclusive Story
Already have an account? Log in
This article is exclusive to STAT+ subscribers
Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts.
Already have an account? Log in
Individual plans
Group plans
View All Plans
To read the rest of this story subscribe to STAT+.
Subscribe
