Alexion has announced that the Food and Drug Administration (FDA) has accepted for review the supplemental Biologics License Application (sBLA) for Soliris (eculizumab) as a possible treatment for patients with refractory generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive; an ultra-rare segment of patients with MG.

The submission is supported by data from the Phase 3 REGAIN study, a randomized, double-blind, placebo-controlled trial which enrolled a total of 125 patients with anti-AChR Abs; full results of the trial have not yet been released. If approved, Soliris would become the first complement inhibitor for patients with refractory AChR-positive gMG. 

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Soliris is already approved to treat paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis, and for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.

“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, PhD, EVP and Global Head of R&D at Alexion. 

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