News

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…

Pregnant women with paroxysmal nocturnal hemoglobinuria (PNH) who receive Soliris (eculizumab) have higher chances of their babies surviving compared with those who don’t, with fewer miscarriages before 24 weeks, a meta-analysis study has found. Biosimilars of Soliris are also available. “Eculizumab appears to be safe and benefits pregnant women with…

Up to 1 in 4 people with paroxysmal nocturnal hemoglobinuria (PNH) who are treated with Ultomiris (ravulizumab-cwvz) or Soliris (eculizumab) experience clinically significant extravascular hemolysis — the destruction of red blood cells — but this isn’t typically associated with severe fatigue, a new global study reported. Soliris…

Treatment with Fabhalta (iptacopan) can ease fatigue and improve quality of life for people with paroxysmal nocturnal hemoglobinuria (PNH), according to data from two Phase 3 clinical trials. “These results … indicate that [Fabhalta] results in clinically meaningful disease control and meaningful improvements in the fatigue and [health-related…