Andrea Lobo, PhD,  —

Note: This story was updated Dec. 6, 2024, to correct that Voydeya is approved as an add-on treatment to standard PNH therapies to help control extravascular hemolysis. The headline was changed to clarify that these were the results of a review study. Voydeya (danicopan) — whether given as a…

Treatment with Empaveli (pegcetacoplan), approved for adults with paroxysmal nocturnal hemoglobinuria (PNH), may lead to clinically meaningful improvements in blood parameters and fatigue among those with impaired bone marrow function. That’s according to a new analysis of data from two Phase 3 clinical trials — PEGASUS (NCT03500549) and…

Bkemv (eculizumab-aeeb) — a biosimilar of Soliris (eculizumab) that was approved in the U.S. earlier this year as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) — shows equivalent safety and efficacy to its reference drug, according to newly published data from a Phase 3 clinical trial. Results…

The specialty pharmacy Onco360 was selected to supply PiaSky (crovalimab-akkz), an antibody-based medication that works to reduce red blood cell destruction, to teenagers and adults with paroxysmal nocturnal hemoglobinuria (PNH). Marketed by Genentech (a Roche subsidiary) in the U.S., the treatment was recently approved by…

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…