Alexion Pharmaceuticals announced that the U.S. Food and Drug Administration (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.
“Given the debilitating impact NMOSD relapses can have on patients and the fact that there is currently no approved therapy, we are committed to getting SOLIRIS to these patients as quickly as possible,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “Based on our strong clinical data, we believe that SOLIRIS can provide significant therapeutic benefits, and we look forward to working with the FDA to facilitate a rapid review.”
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. SOLIRIS has received Orphan Drug Designation (ODD) for the treatment of patients with NMOSD in the U.S., EU and Japan.
Source: Company Press Release