The first approved long-acting complement inhibitor for patients with PNH, ravulizumab, has been approved for nationwide reimbursement by the federal health department. This gives adult PNH patients in Switzerland access to the new treatment standard, which lasts for eight weeks and thus offers patients more private freedom.
Paroxymal nocturnal hemoglobinuria (PNH) is a very rare, complement-mediated, serious disorder that can lead to thrombosis, among a variety of debilitating symptoms and complications. The disease can strike anyone without warning, but predominantly breaks out in the early 30s. Now, the Federal Office of Public Health has approved the first and only long-acting C5 complement inhibitor, ravulizumab (Ultomiris®), for reimbursement nationwide. Adult PNH patients with hemolysis and clinical symptoms indicative of high disease activity, as well as patients who are clinically stable after being treated with eculizumab for at least the past six months, now have access to this new treatment option. The January 2020 Swissmedic approval is based on comprehensive results from two published Phase III studies – the largest Phase III program ever conducted in PNH. In these studies, which included a total of more than 440 patients who were either complement inhibitor-naïve or previously treated with the current standard of care, eculizumab, the efficacy of eight-weekly ravulizumab was shown to be non-inferior to biweekly eculizumab in all eleven endpoints. The safety profile was comparable. Supplemental data published in February 2019 showed that ravulizumab resulted in immediate, complete inhibition of C5 protein, which was maintained over eight weeks, and prevented breakthrough hemolysis due to incomplete C5 inhibition. Ravulizumab has the potential to become the new standard of care, both in complement inhibitor naïve PNH patients and in PNH patients receiving eculizumab.
Source: “Alexion Announces Nationwide Reimbursement of ULTOMIRIS® (Ravulizumab) for the Treatment of Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH),” Sept. 01, 2020, Alexion Pharma GmbH.
InFo ONCOLOGY & HEMATOLOGY 2020; 8(5): 37.
