News

Epysqli (eculizumab-aagh), a Soliris (eculizumab) biosimilar, is now available in the U.S. to reduce red blood cell destruction (hemolysis) in people with paroxysmal nocturnal hemoglobinuria (PNH). A biosimilar contains an active ingredient that’s highly similar to an approved biological medicine, or reference product, with no clinically meaningful differences…

Close monitoring and Empaveli (pegcetacoplan) dose adjustments before and after surgery effectively prevented episodes of breakthrough hemolysis,in a 67-year-old man with paroxysmal nocturnal hemoglobinuria (PNH), according to a case report in Spain. Hemolysis is a hallmark symptom of PNH marked by the breakdown of red blood cells, which…

Paroxysmal nocturnal hemoglobinuria (PNH) was identified as the likely cause of a stroke in a young man, according to a report from India. While high blood pressure and atherosclerosis, a condition marked by the buildup of fatty deposits inside arteries, are commonly associated with stroke, this case “sheds light…

Clinical site activation is now ongoing for a Phase 3 program that will evaluate zaltenibart (OMS906) as a treatment for people with paroxysmal nocturnal hemoglobinuria (PNH), its developer Omeros has announced. A total of 120 sites across 30 countries were chosen for clinical trial participation, according to a…

An unusual meningococcal infection during treatment with Empaveli (pegcetacoplan) was reported in a 16-year-old girl with paroxysmal nocturnal hemoglobinuria (PNH), according to a case report from Finland. Meningococcal infections are a known risk for patients receiving complement inhibitors, such as Empaveli. As such, patients typically are vaccinated before…

People with paroxysmal nocturnal hemoglobinuria (PNH) who switch to Empaveli (pegcetacoplan) after having an inadequate response to other, older treatments usually see good efficacy with the newer therapy, according to a study that looked at real-world data in Italy. Indeed, according to the researchers, “most patients exhibited a…

The use of Fabhalta (iptacopan) — approved in the U.S. in December 2023 as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) — may save about $4 million over a patient’s lifetime compared with intravenous, or into-the-vein, standard of care therapies, according to a study by U.S. researchers. The oral…

Most Chinese adults and adolescents with paroxysmal nocturnal hemoglobinuria (PNH) no longer require blood transfusions after a median of six months of treatment with Soliris (eculizumab). That’s according to a small study at a single center in China, which also found the approved therapy to be safe and…

Health Canada has approved the oral therapy Fabhalta (iptacopan) as a treatment for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have hemolytic anemia. With that decision, Fabhalta has now become the first oral medication that can be used on its own to control PNH to win approval in…

Long-term treatment with Ultomiris (ravulizumab) reduced the risk of death by five times in people with paroxysmal nocturnal hemoglobinuria (PNH), and also tended to eliminate the need for blood transfusions and to improve patients’ quality of life, a new analysis suggests. The findings were detailed in the study,…