BioCryst Secures FDA Orphan Drug Status For Rare Connective Tissue Disease Candidate

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation for BioCryst’s (NASDAQ: BCRX) lead asset BCX9250 for the treatment of fibrodysplasia ossificans progressiva (FOP).

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

Dr. Helen Thackray, Chief research and development officer, commented: "We appreciate the FDA's decision to grant orphan drug designation to BCX9250 as we work toward our goal of bringing this important oral investigational therapy to FOP patients. The benefits available to us through this designation – and the designations we previously received from the FDA and the EMA – have the potential to advance our ALK-2 inhibitor program as efficiently as possible as we move towards beginning trials in FOP patients next year.”

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

The company will present three posters featuring data from BCX9250 Phase 1 program at the American Society for Bone and Mineral Research Annual Meeting, which is being held in Austin, Texas, from September 9-12, 2022.

FDA grants orphan designation to promote the development of a drug that is expected to have significant therapeutic advantage over existing treatments that target a condition affecting 200,000 or fewer U.S. patients annually.

Price Action : BioCryst share are trading around 2 percent at $14.05 on Wednesday during pre-marketing session.