BioCryst’s ALK-2 Inhibitor Granted FDA Orphan Drug Status For Rare Bone Disease

BioCryst Pharmaceuticals has announced that its ALK-2 inhibitor, BCX9250, has been granted Orphan Drug Designation by the US FDA for fibrodysplasia ossificans progressiva (FOP), a rare bone disorder. 

Orphan drugs give developers a number of benefits, including tax credits for clinical trials, exemption from user fees and exclusive marketing rights for up to seven years. 

BCX9250 previously received Fast Track Designation from the FDA. The European Medicines Agency (EMA) has also granted orphan drug status to BCX9250 as well as the PRIME designation, which enables accelerated assessment. 

FOP And ALK-2 Inhibition 

FOP is a very rare genetic disorder characterized by irregular formation of bone outside of the normal skeleton. Bone formation can occur in muscles, tendons and other connective tissues. It could lead to fused joints, restricted movement and premature mortality. FOP is caused by an activating mutation in the activin receptor-like kinase-2 (ALK-2) enzyme which is part of the signaling pathway for normal bone formation. 

An oral drug, BCX9250 is designed to inhibit ALK-2. A Phase 1 trial has indicated that the drug is safe, well tolerated, and has the potential for once-daily dosing. 

BioCryst will present posters featuring preclinical and clinical data on BCX9250, including the aforementioned Phase 1 at the American Society for Bone and Mineral Research Annual Meeting, which is being held in Austin, Texas, from September. 9-12, 2022.

Quick Look At BioCryst

Founded in 1986, BioCryst is based in North Carolina, US. The company focuses on rare diseases, and has two products, berotralstat, a plasma kallikrein inhibitor for hereditary angiodema (HAE) and peramivir injection, an antiviral drug for influenza, approved in the US and internationally. 

Besides BCX9250, BioCryst is investigating a Factor D inhibitor, BCX9930, for complement-mediated diseases such as paroxysmal nocturnal hemoglobinuria (PNH) and an antiviral, galidesivir, for Marburg virus and yellow fever. 

In April, shares in BioCryst took a hit after it said it would pause enrollment for three Phase 2 trials of BCX9930 due to signs of kidney impairment. The company resumed enrollment in August.

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Joy Lin

Without knowledge, there is no imagination. Without imagination, knowledge is meaningless