US FDA Grants Orphan Drug Status To BioCrysts BCX9930 To Treat Paroxysmal Nocturnal Hemoglobinuria

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.

Optimize Your trial insights with Clival Database.

Are you exhausted from the uncertainty of trial insights pricing? Clival Database ensures the clarity in the midst of the global scenario for clinical trials to you.

Clival Database is one of the best databases that offers an outstanding number of clinical trial data in terms of 50,000+ molecules and from primary regulatory markets as well as new entrants like Indian and Chinese markets.

With Clival, you get accurate positioning of historical sales data, patent database, company profiling, safety & efficacy, and prediction of launch of new innovative molecules helping you to align your research and driving down the cost.

To add value, we further break down our analytics for you so that improving your operational effectiveness; optimizing your clinical trials; and offering you accurate and high-quality data at lowest possible prices becomes possible.

Elevate your trial success rate with the cutting-edge insights from Clival database.

Check it out today and make more informed sourcing decisions! Learn More!