Marisa Wexler MS,  —

Chugai Pharmaceutical has applied to regulatory authorities in Japan seeking the approval of crovalimab, an under-the-skin injection therapy that can be administered at home, to treat paroxysmal nocturnal hemoglobinuria (PNH). “We are very pleased that crovalimab, our…

NM3086, an experimental therapy for paroxysmal nocturnal hemoglobinuria (PNH) that’s designed to block the alternative complement pathway’s activation, was generally well tolerated in a first in-human clinical trial. Findings from the Phase 1 study with healthy volunteers suggest NM3086 is working as intended, according to its developer, NovelMed.

Varicella-zoster virus — which causes chickenpox and shingles — can lead to fatal infections in people with paroxysmal nocturnal hemoglobinuria (PNH) who are on immune-suppressing treatment, a case report highlights. Researchers urge clinicians to be aware of signs of possible infection in a report, “Disseminated varicella-zoster…

A bone marrow transplant may be an effective treatment for some people with bone marrow cancer who develop subsequent or co-occurring paroxysmal nocturnal hemoglobinuria (PNH), a study indicates. The study, “Haemolytic paroxysmal nocturnal haemoglobinuria in patients with myeloid neoplasms: A rare association with specific therapeutic implications,”…

A woman with paroxysmal nocturnal hemoglobinuria (PNH) that was being well-controlled with Empaveli (pegcetacoplan) saw her symptoms return after being vaccinated for COVID-19, and then again after contracting COVID-19 itself. According to researchers, the patient experienced breakthrough hemolysis, or a reoccurrence of blood cell destruction, following a COVID-19 vaccination…

Most people with paroxysmal nocturnal hemoglobinuria (PNH) who were treated with Soliris (eculizumab) at a hospital in Germany continued to show signs of disease activity despite treatment, a recent study reports. “The findings of this long-term real-world study suggest that a considerable proportion of patients with PNH treated…

Thrombosis — the serious and potentially life-threatening blood clots that can block blood flow — is a common occurrence in paroxysmal nocturnal hemoglobinuria (PNH) and can affect patients using more recent disease treatments, a study of PNH patients in Greece reported. “Ongoing treatment with complement inhibitors has considerably reduced…

Omeros has launched two Phase 1b clinical trials to test its experimental therapy OMS906 in people with paroxysmal nocturnal hemoglobinuria (PNH). The first trial, which began recruiting late last year, is testing the therapy in people who haven’t been on prior treatment. It has already begun dosing participants. The…

Early screening for paroxysmal nocturnal hemoglobinuria (PNH) and aplastic anemia — two bone marrow disorders that often occur together — is important to prevent acute and serious complications, according to a recent case study. Aplastic anemia affects the stem cells in the bone marrow that give rise to new blood…

People with paroxysmal nocturnal hemoglobinuria (PNH) newly treated with Empaveli (pegcetacoplan) are more likely to see their hemoglobin levels rise and less likely to experience breakthrough disease attacks than those started on Soliris (eculizumab) or Ultomiris (ravulizumab-cwvz). That’s according to a new study reporting the findings from an indirect comparison…