Alexion Announces FDA Approval of ULTOMIRIS® (ravulizumab-cwvz) for Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)

发布时间： 2021-06-10 阅读：484次

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2021.06.07 -- Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced the U.S. Food and Drug Administration (FDA) has approved the expanded use of ULTOMIRIS® (ravulizumab-cwvz) to include children (one month of age and older) and adolescents with paroxysmal nocturnal hemoglobinuria (PNH). ULTOMIRIS, a long-acting C5 inhibitor that offers immediate, complete and sustained complement inhibition, is now the first and only FDA-approved medicine for children and adolescents with PNH.

Since its initial approval in 2018, ULTOMIRIS has quickly become the standard of care in the U.S. for the treatment of adults with PNH. PNH is a complement-mediated disease, which means the symptoms and complications are caused by a lack of regulation, or control, of the complement system, an essential part of the immune system. ULTOMIRIS is designed to target the part of the complement system at the site of disease activity (terminal complement), while preserving function of other parts of the immune system to be able to fight common pathogens and infections. ULTOMIRIS reduces red blood cell destruction in the blood vessels, also known as intravascular hemolysis, and thrombosis (blood clot) risk by providing immediate, complete, and sustained terminal complement inhibition.

This approval is based on interim Phase 3 study results, which showed that ULTOMIRIS was effective in achieving complete C5 complement inhibition through 26 weeks in children and adolescents up to 18 years of age. Additionally, ULTOMIRIS had no reported treatment-related severe adverse events, and no patients discontinued treatment during the primary evaluation period or experienced breakthrough hemolysis, which can lead to disabling or potentially fatal blood clots. The efficacy and safety of ULTOMIRIS in children and adolescents is consistent with the established profile of ULTOMIRIS in clinical studies involving adults with PNH and is representative of the broad PNH patient population seen in the real-world clinical setting.