By Chris Wack


The U.S. Food and Drug Administration has granted Fast Track designation for BioCryst Pharmaceuticals Inc.'s BCX9250 for the prevention of heterotopic ossification in patients with fibrodysplasia ossificans progressiva, the company said Wednesday.

In a Phase 1 clinical trial in healthy subjects, BCX9250 was safe and well tolerated at all doses studied, with linear and dose-proportional exposure supporting the potential for once-daily dosing.

BCX9250 is designed to inhibit the ALK-2 enzyme, which is a part of the normal signaling pathway for bone formation and responds to binding its specific ligands by stimulating normal bone growth and renewal in healthy children and adults. Specific activating mutations of the ALK-2 gene are seen in all cases of FOP.

The purpose of the Fast Track designation is to get important new drugs to patients earlier by facilitating the development, and expediting the review, of drugs to treat serious conditions and fill an unmet medical need. Companies that receive this designation are eligible for benefits including more frequent meetings with and written communication from the FDA, eligibility for accelerated approval and priority review, if relevant criteria are met, and rolling review of the new drug application.


Write to Chris Wack at chris.wack@wsj.com


(END) Dow Jones Newswires

06-08-22 0738ET