Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the Company’s Biologics License Application (BLA) for approval of ALXN1210, the Company’s investigational long-acting C5 complement inhibitor, for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). The FDA set a Prescription Drug User Fee Act (PDUFA) date of February 18, 2019, as part of an expedited eight-month review instead of the standard 12-month review following Alexion’s use of a rare disease priority review voucher. The application is supported by comprehensive data from two rigorous Phase 3 clinical trials.
“We are working with the FDA to facilitate a smooth review,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “Building on comprehensive results from the largest-ever Phase 3 development program in PNH, 11 years of proven efficacy and safety with Soliris®, and 25 years of leadership in complement biology, we are on track with our efforts to establish ALXN1210 as the new standard of care for patients with PNH.”
If approved, ALXN1210 would be the first and only long-acting complement inhibitor for patients with PNH, providing immediate and complete inhibition of the C5 complement protein that is sustained over an eight-week dosing interval. The Phase 3 clinical development program of ALXN1210 is the largest-ever Phase 3 program in PNH. The studies enrolled a very broad and diverse population of more than 440 patients, which included patients who had never been treated with a complement inhibitor and patients who were stable on Soliris® and switched to ALXN1210. Topline data were disclosed in press releases on March 15, 2018 and April 26, 2018, respectively.
In addition to the submission in the U.S. on June 18 and the submission in the European Union (EU) on June 28, Alexion is preparing a submission for a New Drug Application for ALXN1210 as a treatment for patients with PNH in Japan in the second half of the year. The European Medicines Agency (EMA) has accepted and is reviewing the submission for the EU. ALXN1210 has received Orphan Drug Designation (ODD) in the U.S. and EU for the treatment of patients with PNH.
Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, progressive, debilitating, and potentially life-threatening ultra-rare blood disorder that can strike men and women of all races, backgrounds, and ages without warning, with an average age of onset in the early 30s.1,2,3 PNH often goes unrecognized, with delays in diagnosis ranging from one to more than 10 years.2 In patients with PNH, chronic, uncontrolled activation of the complement system, a component of the body’s immune system, results in hemolysis (the destruction of red blood cells)4, which in turn can result in progressive anemia, fatigue, dark urine, and shortness of breath.5,6,7 The most devastating consequence of chronic hemolysis is thrombosis (the formation of blood clots), which can damage vital organs and cause premature death.8 Historically, it had been estimated that one in three patients with PNH did not survive more than five years from the time of diagnosis.2 PNH is more common among patients with disorders of the bone marrow, including aplastic anemia (AA) and myelodysplastic syndromes (MDS).9,10,11 In certain patients with thrombosis of unknown origin, PNH may be an underlying cause.4
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