FDA accepts sBLA for Soliris for treatment of neuromyelitis optica spectrum disorder .- Alexion Pharma

Alexion Pharmaceuticals, Inc.announced that the FDA has accepted for review a supplemental Biologics License Application (sBLA) for the use of Soliris (eculizumab), the company�s first C5 complement inhibitor, as a treatment for patients with neuromyelitis optica spectrum disorder (NMOSD) who have anti-aquaporin-4 (AQP4) auto antibodies.

The FDA granted priority review and set a Prescription Drug User Fee Act (PDUFA) action date of June 28, 2019 . The application is supported by comprehensive data from the successful PREVENT study in patients with anti-AQP4 auto antibody-positive NMOSD.

NMOSD is a rare, devastating, complement-mediated disorder of the central nervous system characterized by relapses. Each relapse results in stepwise accumulation of disability, including blindness and paralysis, and sometimes premature death. Patients who have anti-AQP4 auto-antibodies represent approximately three quarters of all patients with NMOSD. The European Medicines Agency (EMA) is reviewing Alexion�s application to add the treatment of NMOSD to the marketing authorization for Soliris in the European Union (EU), submitted in January 2019 . In addition, Alexion is preparing to submit a supplemental New Drug Application for Soliris in NMOSD in Japan in Q1 2019. .