FDA submission for Uniqure Huntington's drug thrown into question

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.

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