Rare Daily Staff

Alexion Pharmaceuticals reported positive topline results from the from a late-stage study of Soliris in patients with anti-aquaporin-4 (AQP4) auto antibody-positive neuromyelitis optica spectrum disorder (NMOSD).

NMOSD is a rare 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.

There are currently no approved therapies for this disease.

Alexion said the phase 3 study met its primary endpoint of time to first adjudicated on-trial relapse, demonstrating that treatment with Soliris reduced the risk of NMOSD relapse by 94.2 percent compared to placebo. At 48 weeks, 97.9 percent of patients receiving Soliris were free of relapse compared to 63.2 percent of patients receiving placebo. Soliris was generally well tolerated with a safety profile consistent with that seen in previous clinical studies and real-world use in its three approved indications.

“These results far exceeded our expectations. The remarkable reduction in relapse risk demonstrates the unique ability of Soliris to inhibit complement, and suggests a promising new treatment for NMOSD,” said John Orloff, executive vice president and head of research & development at Alexion. “Given that patients currently have no approved therapies, we are moving quickly to discuss these results with regulators and file for approval in the U.S., E.U., and Japan.”

September 24, 2018
Photo: John Orloff, executive vice president and head of research & development at Alexion

 

 

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