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FDA Grants Orphan Drug Designation for BioCryst’s ALK-2 Inhibitor, BCX9250, for the Treatment of Fibrodysplasia Ossificans Progressiva
RESEARCH TRIANGLE PARK, N.C., Aug. 31, 2022 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced the U.S. Food and Drug
About this update from Biocryst Pharmaceuticals, Inc.
[{"type":"text","content":"RESEARCH TRIANGLE PARK, N.C., Aug. 31, 2022 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for BCX9250 for the treatment of fibrodysplasia ossificans progressiva (FOP). The FDA has the option to grant orphan drug designation to applicants for the purpose of supporting the development of a drug or biologic intended to treat a disease or condition that affects fewer than 200,000 individuals in the U.S. Companies that receive this designation are eligible for incentives, including tax credits for qualified clinical trials, exemption from user fees and up to seven years of market exclusivity after approval. “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. FOP is an ultra-rare, severely disabling genetic disorder characterized by heterotopic ossification (HO), or the irregular formation of bone outside the normal skeleton. HO can occur in muscles, tendons, ligaments and other connective tissues. Patients with FOP become bound by this irregular ossification over time, with restricted movement and fused joints, resulting in deformities, restricted mobility and premature mortality. Earlier this year, BioCryst announced that BCX9250 has received Fast Track designation from the FDA, in addition to orphan drug designation and PRIME designation from the European Medicines Agency (EMA). BioCryst will present three posters featuring data from non-clinical studies and first-in-human Phase 1 safety, tolerability and pharmacokinetics study in healthy subjects from the BCX9250 program at the American Society for Bone and Mineral Research Annual Meeting, which is being held in Austin, Texas, from Sept. 9-12, 2022. As the company previously reported in the Phase 1 study, BCX9250 was safe and well tolerated at all doses studied, with linear and dose-proportional e...