Posted by Michael Wonder on 20 Jun 2018
Alexion submits application for priority review and approval of ALXN1210 as a treatment for patients with paroxysmal nocturnal haemoglobinuria in the U.S.
19 June 2018 - Submission in the European Union on track for mid-year and in Japan for the second half of the year.
Alexion Pharmaceuticals today announced the submission of a biologics license application to the U.S. FDA for approval of ALXN1210, the Company’s investigational long-acting C5 complement inhibitor, for the treatment of patients with paroxysmal nocturnal haemoglobinuria (PNH). 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.
