Alexion Pharmaceuticals secures FDA priority review for SOLIRIS in neuromyelitis optica spectrum disorder

Alexion Pharmaceuticals has secured a significant milestone with the FDA granting priority review for its C5 complement inhibitor, SOLIRIS (eculizumab), as a treatment for neuromyelitis optica spectrum disorder (NMOSD) in patients who test positive for anti-aquaporin-4 (AQP4) autoantibodies.

This critical approval comes as part of a supplemental Biologics License Application (sBLA), which has been accepted by the U.S. Food and Drug Administration. The sBLA submission is supported by promising clinical data from the PREVENT study, a trial focused on patients with anti-AQP4 autoantibody-positive NMOSD.

NMOSD is a rare but severe autoimmune disorder characterized by the inflammation of the optic nerve and spinal cord. Patients with NMOSD often face an unpredictable and relapsing course of the disease, which significantly impacts their quality of life and can lead to premature death. Each relapse typically worsens the disability, with optic neuritis often resulting in eye pain or blindness.

Critical Role of Anti-AQP4 Auto-Antibodies in NMOSD

Nearly 75% of NMOSD patients test positive for anti-AQP4 auto-antibodies, which play a critical role in the pathophysiology of the disease. This marks an important subgroup of patients who stand to benefit from the potential approval of SOLIRIS for NMOSD.

John Orloff‘s Statement on FDA Priority Review

John Orloff, Executive Vice President and Head of Research & Development at Alexion Pharmaceuticals, commented on the FDA’s decision, stating, “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.”

Alexion’s Global Expansion Plans for SOLIRIS

This FDA priority review for SOLIRIS is a crucial step toward addressing the unmet need for effective treatments in NMOSD. In addition to its U.S. submission, Alexion Pharmaceuticals also submitted an application to the European Medicines Agency (EMA) last month, seeking approval for SOLIRIS as a treatment for NMOSD. Furthermore, Alexion plans to submit a supplemental New Drug Application (sNDA) in Japan by the first quarter of 2019.

Orphan Drug Designation for SOLIRIS

SOLIRIS has already earned orphan drug designation (ODD) for the treatment of NMOSD in the U.S., European Union, and Japan, reflecting the drug’s potential to serve a rare and underserved patient population.

As the review process continues, all eyes will be on Alexion Pharmaceuticals to see how rapidly the company can bring SOLIRIS to market and provide relief to those suffering from the debilitating effects of NMOSD.