UniQure (QURE) has a clearer path toward possible accelerated approval of its investigational drug for Huntington's disease after the US Food and Drug Administration signaled it is open to accepting a biologics license application based on three-year data, RBC Capital Markets said in a note Wednesday.
The investment firm said the regulator will likely accept a BLA with priority review when the company files it in Q3 because of the unmet need.
The FDA's willingness to consider the filing without waiting for four-year data and placebo-controlled trial signals regulatory leniency that is also "great news" for the broader industry and improves the outlook for the drug, according to the note.
Analysts said they now estimate a 75% chance of approval, compared with 50% previously, although the company still needs to reach alignment with the FDA on the confirmatory trial details prior to BLA submission.
RBC has an outperform, speculative risk rating on the stock, with a price target raised to $65 from $35.
Shares of uniQure were down more than 2% in Thursday trading.
Price: 46.91, Change: -1.25, Percent Change: -2.60
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