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Alexion Submits BLA For Priority Review And Approval Of ALXN1210

Alexion Pharmaceuticals, Inc. (ALXN) submitted on Tuesday a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for approval of ALXN1210, the Company's investigational long-acting C5 complement inhibitor, for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH).

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.

The submission uses a rare disease priority review voucher, which designates the BLA for an expedited eight-month review by the FDA instead of the standard 12-month review.

The application is supported by comprehensive data from two rigorous Phase 3 clinical trials in the largest population of patients with PNH ever studied: more than 440 patients, which included patients who had never received a complement inhibitor, and patients who were stable on Soliris (eculizumab) and switched to ALXN1210.

Weight-optimized treatment with ALXN1210 every eight weeks demonstrated non-inferiority to treatment every two weeks with Soliris on all primary endpoints and key secondary endpoints in both studies.

ALXN1210 has received Orphan Drug Designation (ODD) for the treatment of patients with PNH in the U.S. and EU, and for the subcutaneous treatment of patients with atypical hemolytic uremic syndrome (aHUS) in the U.S.

In addition to the BLA in the U.S., Alexion is preparing submissions for the approval of ALXN1210 as a treatment for patients with PNH in the European Union (EU) by mid-year and in Japan in the second half of the year.

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