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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…

Paroxysmal nocturnal hemoglobinuria (PNH) is being diagnosed more frequently in Italy, and while treatment with complement inhibitors can be effective, it carries a high yearly cost of over €133,000 (about $155,000) per patient, mostly due to medication expenses. According to a study, every fourth patient received treatment…

Six months of Fabhalta (iptacopan) raised hemoglobin levels, eliminated the need for blood transfusions, and eased fatigue in most adults with paroxysmal nocturnal hemoglobinuria (PNH) who switched from standard anti-C5 therapies. That’s according to data from the open-label Phase 3b APPULSE-PNH (NCT05630001) that evaluated the therapy’s efficacy and…

Health Canada has approved Roche’s PiaSky (crovalimab) for treating paroxysmal nocturnal hemoglobinuria (PNH) in adults and adolescents, ages 13 and older, who weigh at least 40 kg (88 pounds). The treatment is the first one approved in Canada that’s administered via monthly injections under the skin, or subcutaneously,…

One year of treatment with Fabhalta (iptacopan) boosts hemoglobin levels, eliminates the need for blood transfusions, and eases fatigue in most adults with paroxysmal nocturnal hemoglobinuria (PNH). That’s according to final data from two global Phase 3 clinical trials: APPLY-PNH (NCT04558918) and APPOINT-PNH (NCT04820530), which evaluated…

Novelmed Therapeutics’ ruxoprubart prevented red blood cell destruction in treatment-naïve adults with paroxysmal nocturnal hemoglobinuria (PNH), allowing these untreated patients to avoid blood transfusions altogether. That’s according to 12-week interim data from an ongoing Phase 2 clinical trial (NCT05646524), which showed that the PNH treatment, when administered…