News

Ultomiris (ravulizumab-cwvz) is as effective or better than Soliris (eculizumab) at keeping paroxysmal nocturnal hemoglobinuria (PNH) under control, a real-world case series shows. “Our real-world data reflect routine clinical practice, supporting the notion that [Ultomiris] may provide enhanced disease stability in high-risk patients,” researchers wrote. The results were…

Most people with paroxysmal nocturnal hemoglobinuria (PNH) treated with Fabhalta (iptacopan) in clinical trials reported high satisfaction and a meaningful easing of symptoms with the oral therapy, and generally preferred it over other older treatments. In fact, all patients previously treated with the PNH therapies Soliris (eculizumab)…

Atropos Health will partner with Novartis to develop artificial intelligence (AI) models to help identify people with rare diseases, such as paroxysmal nocturnal hemoglobinuria (PNH), who haven’t yet been diagnosed. Using AI algorithms trained on real-world data, the collaboration seeks to reduce the time from the onset…

An experimental combination of pozelimab and cemdisiran was found to be safe in adults with paroxysmal nocturnal hemoglobinuria (PNH) and to keep hemolysis under control — even with less frequent dosing, according to a study. Hemolysis is the premature breakdown of red blood cells. The Phase 2 clinical…

In interviews, people in Japan with paroxysmal nocturnal hemoglobinuria (PNH) reported financial and logistical concerns — primarily, losing time from work and spending hours waiting for examinations and infusions — that increased the treatment burden they face in living with PNH. Patients also shared with researchers their worries about…

Over the course of up to three years of treatment with Empaveli (pegcetacoplan), most people with paroxysmal nocturnal hemoglobinuria (PNH) did not require blood transfusions, according to a new analysis of clinical trial data. The analysis also indicated Empaveli led to long-term easing of fatigue and reductions in…

About one in five patients newly diagnosed with paroxysmal nocturnal hemoglobinuria (PNH) in the Nordic countries has a history of blood clots obstructing blood vessels — known as thrombosis — or develops kidney failure, a study in Denmark, Finland, Norway, and Sweden found. “The high number of thromboses and…

Treatment with HRS-5965, a complement inhibitor being developed by Jiangsu Hengrui Pharmaceuticals, increased the levels of hemoglobin and reduced red blood cell destruction in people with paroxysmal nocturnal hemoglobinuria (PNH), according to data from a clinical trial. Data from the Phase 2 trial (NCT06051357), which assessed the…

Direct oral anticoagulants, or DOACs, are safe and effective for preventing recurrent venous thromboembolism (VTE), or blood clots forming in a vein, in people with paroxysmal nocturnal hemoglobinuria (PNH), a study reports. These medications, given after standard vitamin K antagonists (VKAs) and low molecular weight heparin (LMWH) during the…

A teenage girl with paroxysmal nocturnal hemoglobinuria (PNH) who experienced extravascular hemolysis, which occurs when red blood cells are destroyed outside of the blood vessels, saw her condition significantly improve after starting on Empaveli (pegcetacoplan) — a treatment currently approved only for adults with PNH. That’s according to…