BioCryst Gets FDA Fast Track Designation For Bone Formation Disorder Candidate

The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to BioCryst Pharmaceuticals's BCRX lead drug candidate BCX9250 for the prevention of heterotopic ossification (HO) in patients with fibrodysplasia ossificans progressiva (FOP).

Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare, severely disabling genetic disorder characterized by HO, or the irregular formation of bone outside the normal skeleton.

BCX9250 was safe and well tolerated at all doses studied, In a Phase 1 clinical trial in healthy subjects.

Helen Thackray, chief research and development officer, commented : "We are pleased with the FDA's decision to grant Fast Track designation to BCX9250, as there is a significant unmet need among patients living with FOP. With this designation following the EMA's recent decision to grant PRIME eligibility for BCX9250 in Europe, we believe the non-clinical data and the first-in-human Phase 1 safety, tolerability and pharmacokinetics study in healthy subjects support the potential of our ALK-2 inhibitor program to be a meaningful therapeutic advance for the FOP community."

FDA Fast Track Designation is intended to expedite the development and review of new drugs to treat serious medical conditions that fill unmet medical needs.

BioCryst Pharmaceuticals shares are currently trading higher 4 percent at $10.79.