FDA Accepts sBLA Filing of Soliris® as a Potential Treatment for Patients with Refractory Generalize

Alexion Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Company’s supplemental Biologics License Application (sBLA) to extend the indication for Soliris® (eculizumab) as a potential treatment for patients with refractory generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. The sBLA submission is supported by comprehensive data from the Phase 3 REGAIN study. The FDA set a Prescription Drug User Fee Act (PDUFA) date of October 23, 2017.

"We look forward to working with the FDA to bring this potentially life-transforming treatment to patients who are in dire and urgent need of effective treatment," said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion. "Refractory gMG is an ultra-rare disease. Despite existing treatment options for gMG, patients with refractory gMG continue to face severe complications, including difficulty walking, talking, swallowing, and breathing normally. Exacerbations of their disease may be life-threatening and require hospitalization and intensive care."

If approved, Soliris would be the first and only complement inhibitor for patients with refractory AChR-positive gMG. Soliris has received Orphan Drug Designation (ODD) for the treatment of patients with MG in the U.S. and EU. Soliris is not approved in any country for the treatment of patients with refractory AChR-positive gMG.