EMA committee separately recommended approval of drug for same indication in Europe.
FDA approved Alexion Pharmaceuticals’ Soliris™ (eculizumab) for the treatment of pediatric and adult patients with the genetic disorder atypical hemolytic uremic syndrome (aHUS). The firm separately reported that the EMA’s European Committee for Medicinal Products for Human Use had recommended approval of the drug for the same indication. Soliris is currently indicated in the U.S., EU, Japan, and other markets for the treatment of paroxysmal nocturnal hemoglubinuria (PNH).
Soliris is a terminal complement inhibitor that targets uncontrolled complement activation to inhibit complement-mediated thrombotic microangiopathy (TMA), which leads to disease-related thrombosis, renal impairment, seizures, and angina in aHUS patients. Clearance in the U.S. for the aHUS indication was granted under FDA’s accelerated approval process, based on TMA outcomes from two prospective, pivotal Phase II open-label studies in adult and adolescent patients, and a retrospective study in adult, adolescent, and pediatric patients.
Data from two the two studies in adolescents and adults showed treatment using Soliris resulted in hematologic normalization in 76–90% of patients, and led to TMA event-free status in 80–87% of patients. Treated individuals also showed statistically significant reductions in TMA intervention rates, along with maintained or improved kidney function, reduced need for dialysis, and improved quality of life.
Data from pediatric patients in the third study showed that Soliris therapy led to normalization of platelet counts in 89% of treated patients, along with a reduction in the TMA intervention rate. None of the patients required new dialysis during treatment with the drug.
Prospective studies in additional patients are ongoing to confirm the benefits of Soliris against aHUS. The drug is also being evaluated for the treatment of patients with shiga toxin E. coli-related hemolytic uremic syndrome. Soliris is separately being evaluated either in house, or by independent investigators, for the treatment of dense deposit disease, myasthenia gravis, multifocal motor neuropathy, and for use in patients at high risk of kidney transplant rejection. Alexion says it is also planning studies to evaluate Soliris in patients with the blood cell disorders catastrophic antiphospholipid syndrome and cold agglutinin disease.