Alexion announced that the Food and Drug Administration (FDA) has approved Soliris (eculizumab) as a treatment for adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody-positive.
The FDA approval was based on data from the Phase 3, multicenter, randomized, double-blind, placebo-controlled REGAIN study that randomized 62 patients to Soliris and 63 to placebo. The primary efficacy endpoint for gMG was a comparison of the change from baseline between treatment groups in the Myasthenia Gravis-Specific Activities of Daily Living scale (MG-ADL) total score at Week 26. Treatment with Soliris showed a statistically significant difference in the mean change from baseline to Week 26 in MG-ADL total scores (–4.2 points vs. –2.3 points; P=0.006).
Soliris, a complement inhibitor, is already approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis, and for the treatment of atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy. Its precise mechanism in gMG patients is unknown, but is presumed to involve reduction of terminal complement complex C5b-9 deposition at the neuromuscular junction.
Soliris is only available under a Risk Evaluation and Mitigation Strategy (REMS) program that prescribers are required to enroll in. The product is supplied in 30mL single-dose vials containing 10mg/mL strength preservative-free solution.
Alexion also offers a personalized patient support program called OneSource staffed with nurse case managers that can assist with health insurance navigation, disease information, continuity of care, and community connections.
For more information call (888) 765-4747 or visit Soliris.net.