Alexion aims for fourth rare disease use for blockbuster Soliris
Pharmaphorum Media Limited | February 26, 2019
The FDA has granted a fast review for Alexion Pharmaceuticals’ filing for its Soliris, as a treatment for a rare neurological disease that leads to worsening disability, blindness, paralysis, and possibly early death. In a statement, Alexion said the FDA has accepted its filing for Soliris (eculizumab), its C5 complement inhibitor, as a treatment for patients with neuromyelitis optica spectrum disorder (NMOSD), who have anti-aquaporin (AQP4) autoantibodies. The FDA is due to make a decision by June 28, Alexion said. Alexion said the filing is supported by data from the PREVENT study in patients with anti-AQP4 autoantibody-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.