Alexion Pharmaceuticals has bagged FDA priority review for the use of its C5 complement inhibitor SOLIRIS (eculizumab) as a treatment of neuromyelitis optica spectrum disorder (NMOSD) in patients having anti-aquaporin-4 (AQP4) auto antibodies.
The US pharma company’s supplemental Biologics License Application (sBLA), which has been accepted by the FDA, is backed by data from the PREVENT study in patients with anti-AQP4 auto antibody-positive NMOSD.
A rare relapsing autoimmune disorder, NMOSD selectively leads to inflammation in the optic nerve and spinal cord. NMOSD patients face an unpredictable, relapsing, and deteriorating course of disease with each relapse increasing disability, and possibly resulting in premature death. Optic neuritis can lead to eye pain and blindness.
Patients with anti-AQP4 auto-antibodies account to nearly three quarters of all patients with NMOSD.
Commenting on the FDA priority review of SOLIRIS, John Orloff – Executive Vice President and Head of Research & Development at Alexion Pharmaceuticals, said: “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.
“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.”
Last month, Alexion Pharmaceuticals submitted an application to the European Medicines Agency (EMA) seeking SOLIRIS approval for NMOSD treatment. The US pharma company is also looking to submit a supplemental New Drug Application in Japan in Q1 2019 for the C5 complement inhibitor for NMOSD treatment.
SOLIRIS has orphan drug designation (ODD) for the treatment of NMOSD in the US, European Union and Japan.