FDA Approves Myasthenia Gravis Treatment
Soliris is only available under a Risk Evaluation and Mitigation Strategy program.
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 =.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.
FDA approves Soliris® (eculizumab) for the treatment of patients with generalized myasthenia gravis (GMG) [news release]. New Haven, CT: Alexion; October 23, 2017. Accessed October 25 2017.