Margarida Maia, PhD,  —

Fatigue affects the ability of adults with paroxysmal nocturnal hemoglobinuria (PNH) to perform daily tasks, interact socially, and stay focused, even with treatment, showing how the disease limits physical, emotional, and social well-being, according to a real-world study. The findings indicate how the impact on health-related quality of life…

A novel combination of pozelimab and cemdisiran appears to be better than Ultomiris (ravulizumab) at controlling intravascular hemolysis — the rupture of red blood cells inside blood vessels — in adults with paroxysmal nocturnal hemoglobinuria (PNH), showing promise as a potential treatment for the rare acquired disease. That’s…

The European Commission has approved Roche’s PiaSky (crovalimab) to treat adults and adolescents with paroxysmal nocturnal hemoglobinuria (PNH), ages 12 and older who weigh at least 40 kilograms (88 pounds). PiaSky is the first treatment for PNH in the European Union that’s administered subcutaneously (under the skin) once a…

Using Empaveli (pegcetacoplan) during a new pregnancy led to a healthy outcome for a mother and her baby boy, even though complications of paroxysmal nocturnal hemoglobinuria (PNH) arose. The mother, who previously had two miscarriages and a poor response to Soliris (eculizumab), recovered from an episode of…

Most of the adults with paroxysmal nocturnal hemoglobinuria (PNH) who switched from Soliris (eculizumab) to PiaSky (crovalimab) in a clinical trial said they preferred PiaSky because it was easier to use, required fewer hospital visits, and took less time to administer. That’s according to data from…

Individualized dosing of Empaveli (pegcetacoplan) may allow for a larger interval between doses in about 30% of adults with paroxysmal nocturnal hemoglobinuria (PNH), while cutting costs by about 8%, according to a study that simulated a clinical trial. The study, “Development of a target concentration intervention to…

An independent appraisal committee voted that there was adequate evidence to show that adding danicopan to a C5 inhibitor may benefit certain people with paroxysmal nocturnal hemoglobinuria (PNH), pending price listing. Committee members also decided the evidence isn’t adequate to show a net health benefit for Fabhalta (iptacopan)…

People in Japan using C5 inhibitors to treat their paroxysmal nocturnal hemoglobinuria (PNH) continue to experience symptoms like fatigue and shortness of breath, even if their hemoglobin levels surpass the median of real-world PNH populations, a survey study reports. “This study contributes to understanding the unmet needs of the…

Almost 6 out of 10 people with paroxysmal nocturnal hemoglobinuria (PNH) living in the U.S. may be being given a higher initial loading dose of Ultomiris (ravulizumab) than is recommended on the medication’s label, a database study into its clinical use reports. Researchers also observed that maintenance doses,…

Danicopan, an experimental oral medication, continued to help control anemia when taken for up to 48 weeks by adults with paroxysmal nocturnal hemoglobinuria (PNH) who experience a breakdown of red blood cells outside of blood vessels — called extravascular hemolysis — despite being on a C5 inhibitor. That’s according…