FDA Grants Fast Track Designation for BioCryst’s ALK-2 Inhibitor, BCX9250
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for BCX9250 for the prevention of heterotopic ossification (HO) in patients with fibrodysplasia ossificans progressiva (FOP).
According to the FDA, 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 (NDA).
“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,” said Dr. Helen Thackray, chief research and development officer of BioCryst.
FOP is an ultra-rare, severely disabling genetic disorder characterized by 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.
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 (bone morphogenic proteins, BMPs) 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. An activating mutation in ALK-2 is necessary for the disease to occur, making the ALK-2 enzyme an ideal drug target for treatment of FOP.
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.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO® (berotralstat) is approved in the United States and multiple global markets. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and yellow fever. RAPIVAB® (peramivir injection) is approved in the U.S. and multiple global markets, with post-marketing commitments ongoing.
June 10, 2022
