BioCryst Pharmaceuticals announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation for its oral Factor D inhibitor, BCX9930, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).According to the FDA, the Orphan Drug Designation Program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the US.Orphan Drug designation qualifies BCX9930 for various development incentives, including tax credits for certain clinical costs, a waiver of the new drug application fee and a designated period of market exclusivity following approval.In addition to the Orphan Drug designation, the FDA also has granted Fast Track designation for BCX9930 in PNH.“As an oral Factor D inhibitor monotherapy for PNH patients, BCX9930 would address a significant unmet medical need and we look forward to our discussions later this year with the FDA to inform how we advance this important medicine to patients with PNH and other complement-mediated diseases as quickly as possible,” said Dr. William Sheridan, chief medical officer of BioCryst.Paroxysmal nocturnal hemoglobinuria (PNH), is a rare, serious, and potentially life-threatening complement-mediated blood disease that causes red blood cells to rupture. The complement system is part of the body’s natural immune system and is responsible for helping the body eliminate microbes and damaged cells. Once activated, the complement system stimulates inflammation, phagocytosis and cell lysis. Excessive or uncontrolled activation of the complement system can cause severe, and potentially fatal, immune and inflammatory disorders, such as PNH.
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