News

European regulators have recommended approving Epysqli, a Soliris (eculizumab) biosimilar, to treat children and adults with paroxysmal nocturnal hemoglobinuria (PNH). The recommendation came from the Committee for Medicinal Products for Human Use (CHMP) and will serve as the basis for a final approval by the European Commission. Epysqli…

Most people with paroxysmal nocturnal hemoglobinuria (PNH) who were treated with Soliris (eculizumab) at a hospital in Germany continued to show signs of disease activity despite treatment, a recent study reports. “The findings of this long-term real-world study suggest that a considerable proportion of patients with PNH treated…

Thrombosis — the serious and potentially life-threatening blood clots that can block blood flow — is a common occurrence in paroxysmal nocturnal hemoglobinuria (PNH) and can affect patients using more recent disease treatments, a study of PNH patients in Greece reported. “Ongoing treatment with complement inhibitors has considerably reduced…

Omeros has launched two Phase 1b clinical trials to test its experimental therapy OMS906 in people with paroxysmal nocturnal hemoglobinuria (PNH). The first trial, which began recruiting late last year, is testing the therapy in people who haven’t been on prior treatment. It has already begun dosing participants. The…

Early screening for paroxysmal nocturnal hemoglobinuria (PNH) and aplastic anemia — two bone marrow disorders that often occur together — is important to prevent acute and serious complications, according to a recent case study. Aplastic anemia affects the stem cells in the bone marrow that give rise to new blood…

People with paroxysmal nocturnal hemoglobinuria (PNH) newly treated with Empaveli (pegcetacoplan) are more likely to see their hemoglobin levels rise and less likely to experience breakthrough disease attacks than those started on Soliris (eculizumab) or Ultomiris (ravulizumab-cwvz). That’s according to a new study reporting the findings from an indirect comparison…

ARO-C3, an experimental treatment being developed for paroxysmal nocturnal hemoglobinuria (PNH) and other disorders mediated by the complement system, appears to be working as intended in a group of healthy volunteers. That’s according to interim data from an ongoing Phase 1/2 trial testing the Arrowhead Pharmaceuticals therapy in adults…

Most people with paroxysmal nocturnal hemoglobinuria (PNH) benefited from Soliris (eculizumab) therapy when they adhered to strict treatment guidelines, a real-world Dutch study showed. Because most had an incomplete response, new therapies are needed to improve real-world outcomes, however, the study’s researchers suggested, and emphasized that their data could…