News

The management approach to paroxysmal nocturnal hemoglobinuria (PNH) in the Middle East is generally consistent with that reported in other regions, according to a review of published studies. Across all studies from Saudi Arabia, Kuwait, Egypt, Oman, and Lebanon, anemia and fatigue were common presenting symptoms, with most patients…

Chinese regulators have given the green light to Argo Biopharmaceutical to launch a Phase 2 clinical trial testing its treatment candidate BW-40202 in people with paroxysmal nocturnal hemoglobinuria (PNH). The trial is expected to begin in January 2026, Argo said in a company press release announcing the decision…

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…