News

A single dose of Homology Medicines’ gene therapy candidate HMI-104 led to the sustained production of a therapeutic antibody to treat paroxysmal nocturnal hemoglobinuria (PNH), preclinical studies suggest. “Given the severity of PNH, which is a rare, acquired blood disorder, and the unmet need associated with chronically administered…

Iptacopan, an experimental oral medication being developed by Novartis, increased hemoglobin to near-normal levels in people with paroxysmal nocturnal hemoglobinuria (PNH) — without the need for blood transfusions — according to data from two global Phase 3 clinical trials. The trials, APPLY-PNH (NCT04558918) and APPOINT-PNH (NCT04820530),…

An experimental therapy being developed by Omeros can nearly double the level of hemoglobin safely after three doses in severely anemic people with paroxysmal nocturnal hemoglobinuria (PNH). That’s according to interim data from an ongoing Phase 1b clinical trial that’s evaluating the effect of once-monthly subcutaneous (under-the-skin) injections…

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…

European regulators have recommended approving Epysqli, a Soliris (eculizumab) biosimilar, to treat children and adults with paroxysmal nocturnal hemoglobinuria (PNH). The recommendation came from the Committee for Medicinal Products for Human Use (CHMP) and will serve as the basis for a final approval by the European Commission. Epysqli…

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…