News

Levels of certain immune proteins in the blood may help predict how well people with paroxysmal nocturnal hemoglobinuria (PNH) respond to the therapies Soliris (eculizumab) and PiaSky (crovalimab-akkz), a new study reports. “These findings indicate that higher baseline levels of these markers may help identify patients at…

Vemircopan, a therapy that Alexion, AstraZeneca Rare Disease was developing for paroxysmal nocturnal hemoglobinuria (PNH), increased hemoglobin levels in a Phase 2 study, but frequent episodes of hemolysis — the destruction of red blood cells — were noted.  In a…

Despite therapy with complement inhibitors, half of individuals with paroxysmal nocturnal hemoglobinuria (PNH) develop breakthrough hemolysis, or BTH — when PNH symptoms suddenly return despite ongoing treatment — according to a real-world study. The incidence and severity of BTH were found to be particularly high in patients treated with…

Novo Nordisk has entered a license agreement with Omeros for the development and commercialization of zaltenibart (OMS906), an experimental treatment for paroxysmal nocturnal hemoglobinuria (PNH). The deal, expected to be close by year-end 2025, will give Novo Nordisk exclusive global rights for zaltenibart in all indications. Once finalized,…

People switching between treatments for paroxysmal nocturnal hemoglobinuria (PNH) may develop transient immune complex reactions (TICRs) — a type of inflammatory reaction — but these are usually not serious and manageable, a new analysis of more than 200 people finds. The analysis covered people who switched from Soliris…

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…