FDA Approves Soliris (eculizumab) for Generalized Myasthenia Gravis and NMOSD

Soliris (eculizumab) is a complement inhibitor. It binds to complement protein C5, inhibiting its cleavage to C5a and C5b, thereby preventing the formation of the membrane attack complex (MAC). This reduces complement-mediated cell destruction and thrombotic microangiopathy.

Soliris is indicated for: - Paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. - Atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy. Limitation of use: Not indicated for Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). - Generalized myasthenia gravis (gMG) in adult and pediatric patients six years of age and older who are anti-acetylcholine receptor (AChR) antibody positive. - Neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.

For intravenous infusion only. - PNH (adults 18+): 600 mg weekly for 4 weeks, then 900 mg at week 5, then 900 mg every 2 weeks. - aHUS, gMG, NMOSD (adults 18+): 900 mg weekly for 4 weeks, then 1200 mg at week 5, then 1200 mg every 2 weeks. - Pediatric aHUS (<18 years) and gMG (6 years and older): Weight-based dosing (see Table 1 in label). - Supplemental dosing required after plasmapheresis/plasma exchange or fresh frozen plasma infusion (aHUS, gMG, NMOSD) and with IVIg (gMG). - Vaccinate against meningococcal infection at least 2 weeks prior to initiation; if urgent, provide antibacterial prophylaxis and vaccinate as soon as possible. - Prescribers must enroll in the ULTOMIRIS and SOLIRIS REMS.

Trial data not available in label.

- Boxed warning: Serious meningococcal infections. Vaccinate patients against meningococcal serogroups A, C, W, Y and B at least 2 weeks prior to initiation. If urgent therapy needed, provide antibacterial prophylaxis and vaccinate as soon as possible. - Enrollment in REMS required. - Administer at recommended time points or within two days.

Soliris is a targeted complement inhibitor for rare, complement-mediated disorders. It is indicated for PNH, aHUS, anti-AChR antibody-positive gMG, and anti-AQP4 antibody-positive NMOSD. It is not indicated for STEC-HUS. Use requires meningococcal vaccination and REMS enrollment.