Alexion Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved Ultomiris (ravulizumab-cwvz), the first and only long-acting C5 complement inhibitor administered every eight weeks, for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating ultra-rare blood disorder characterized by complement-mediated destruction of the red blood cells (hemolysis).
PNH can cause a wide range of debilitating symptoms and complications, including thrombosis, which can occur throughout the body, and result in organ damage and premature death.1,2,3,4,5,6,7,8
The approval of Ultomiris comes ahead of the Prescription Drug User Fee Act (PDUFA) date of February 18, 2019, set by the FDA as part of an expedited eight month review following Alexion’s use of a rare disease priority review voucher.
It is based on comprehensive results from two Phase 3 studies, which were recently published in Blood.9,10 In those studies, which included 441 patients who had either never been treated with a complement inhibitor before, or who had been stable on Soliris, the efficacy of Ultomiris administered every eight weeks was non-inferior to the efficacy of Soliris administered every two weeks on all 11 endpoints.
The safety profile of Ultomiris was similar to that of Soliris. Recently presented additional data showed that Ultomiris provided immediate and complete C5 inhibition that was sustained for eight weeks,11 and that Ultomiris eliminated breakthrough hemolysis associated with incomplete C5 inhibition.12 The entire clinical development program for Ultomiris to date represents more than 600 patient years of experience.
Regulatory authorities in the European Union (EU) and Japan have accepted and are reviewing applications for the approval of Ultomiris as a treatment for adults with PNH. Alexion continues to work with the authorities to enable timely reviews.
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9 Lee JW, Sicre de Fontbrune F, Lee LWL et al. [published online ahead of print, December 3, 2018]. Blood. doi:10.1182/blood-2018-09-876136.
10 Kulasekararaj AG, Hill A, Rottinghaus ST et al. [published online ahead of print December 3, 2018]. Blood. doi:10.1182/blood-2018-09-876805.
11 Peffault de Latour R, Brodsky RA, Ortiz S et al. American Society of Hematology (ASH) Annual Meeting, San Diego, December 2, 2018;Session 101:2330.
12 Brodsky RA, Peffault de Latour R, Rottinghaus ST et al. American Society of Hematology (ASH) Annual Meeting, San Diego, December 3, 2018;Session 101:626.
(Source: Alexion Pharmaceuticals, Inc.)