The U.S. Food and Drug Administration has approved a new drug called Epysqli (eculizumab-sbja) for three rare conditions: paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and generalized myasthenia gravis (gMG) in adults who test positive for anti-acetylcholine receptor antibodies. Epysqli is a complement inhibitor given as an intravenous infusion. It works by blocking part of the immune system that can damage blood cells and nerves.
For PNH, the typical adult dose is 600 mg once a week for 4 weeks, then 900 mg at week 5, followed by 900 mg every 2 weeks. For aHUS and gMG in adults, the dose is 900 mg weekly for 4 weeks, then 1200 mg at week 5, then 1200 mg every 2 weeks. For children with aHUS, the dose is based on weight.
This approval provides an additional treatment option for people living with these serious conditions. It does not treat Shiga toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). If you or a loved one has one of these conditions, talk to your doctor about whether Epysqli might be right for you.