Alexion Seeks Eculizumab Approval for Ultra-Rare Neuromuscular Disease

If approved for this indication Soliris would become the first complement inhibitor for this patient population
If approved for this indication Soliris would become the first complement inhibitor for this patient population

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. 

For more information visit Alexion.com.