FDA approved Epysqli (eculizumab-sbja) for PNH, aHUS, and gMGFDA approved new drug Epysqli for three rare blood and nerve conditions.

Epysqli 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 lysis and thrombotic microangiopathy.

Epysqli is indicated for: - Treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. - Treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy. - Treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. Limitation of Use: Not indicated for Shiga toxin E. coli-related hemolytic uremic syndrome (STEC-HUS).

For intravenous infusion only. Vaccinate against meningococcal infection at least 2 weeks prior to initiation. Enroll in the EPYSQLI REMS. - PNH (adults): 600 mg weekly for first 4 weeks, then 900 mg at week 5, then 900 mg every 2 weeks. - aHUS and gMG (adults): 900 mg weekly for first 4 weeks, then 1200 mg at week 5, then 1200 mg every 2 weeks. - aHUS (pediatric): Weight-based dosing (see label Table 1). Supplemental dosing required with plasmapheresis/plasma exchange/fresh frozen plasma infusion (aHUS, gMG) and with IVIg (gMG).

Not reported in label.

Warnings: Risk of meningococcal infection; vaccinate according to ACIP recommendations. If urgent therapy needed, provide antibacterial prophylaxis and vaccinate as soon as possible. Must enroll in REMS. Administer within 2 days of scheduled time points. Contraindications: Not reported in label.

Epysqli is a complement inhibitor approved for PNH, aHUS, and anti-AChR antibody-positive gMG. It is not indicated for STEC-HUS. It offers an alternative to existing complement inhibitors for these conditions. Clinicians should consider vaccination and REMS requirements before initiating therapy.