Andrea Lobo, PhD,  —

A 43-year-old woman with paroxysmal nocturnal hemoglobinuria (PNH) who experienced extravascular hemolysis — which occurs when red blood cells are destroyed or broken down outside of the blood vessels — despite previous treatment saw her condition significantly improve after initiating treatment with Fabhalta (iptacopan), according to a case…

The U.S. Food and Drug Administration (FDA) has approved Bkemv (eculizumab-aeeb), the first interchangeable biosimilar of Soliris (eculizumab), to treat people with paroxysmal nocturnal hemoglobinuria (PNH). Soliris is an intravenous (into-the-vein) antibody-based therapy that is approved to reduce hemolysis, or red blood cell destruction, in PNH patients. As…

An intensive dosing regimen of Empaveli (pegcetacoplan) effectively managed events of acute red blood cell destruction, called hemolysis, in people with paroxysmal nocturnal hemoglobinuria (PNH), according to an ongoing clinical study’s interim data. Also, the intensive regimen was found to be safe and well tolerated in these patients,…

Dawn Health, a Danish digital health company, and Novartis have launched a smartphone app designed to help people living with paroxysmal nocturnal hemoglobinuria (PNH) with disease management. The free Ekiva PNH app helps patients track their symptoms and prepare for medical appointments, while also providing a…

The U.S. Food and Drug Administration (FDA) has given orphan drug designation to NM5072, an experimental treatment for anemia in people with paroxysmal nocturnal hemoglobinuria (PNH). The potential therapy showed safety and tolerability in an initial clinical trial in healthy volunteers, and a Phase 2 trial in PNH patients…

NM8074, an investigational antibody therapy for paroxysmal nocturnal hemoglobinuria (PNH), will now be called ruxoprubart, its developer NovelMed has announced. The name change came after the USAN, or United States Adopted Names Council, selected ruxoprubart as the unique generic, or nonproprietary, name for the therapy candidate. According to…

Treatment with Empaveli (pegcetacoplan) — a C3 protein blocker — effectively improved disease control and health-related quality of life, while reducing fatigue in people with paroxysmal nocturnal hemoglobinuria (PNH) in the U.S. and Europe, a real-world study reported. Study findings also showed higher physician and patient satisfaction with…