BioCryst Pharmaceuticals Granted Orphan Drug Designation from U.S. FDA for BCX9250

On August 31, 2022, BioCryst Pharmaceuticals announced that they have been granted orphan drug designation from the U.S. Food & Drug Administration (FDA) for their investigational drug to treat FOP, BCX9250.

From BioCryst's press release, “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,” said Dr. Helen Thackray, chief research and development officer of BioCryst.

BioCryst has previously completed a phase 1 trial of BCX9250 in healthy humans. Congratulations to BioCryst Pharmaceuticals for achieving this important milestone!

Learn more about orphan drug designation and read the full press release.