Omeros’ zaltenibart (OMS906) as a single therapy has shown promise as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) in a small clinical study, normalizing signs of hemolysis, or red blood cell destruction, in adults with hard-to-treat disease, according to new interim data. The trial enrolled PNH patients who…
Treatment with Ultomiris (ravulizumab) successfully reduced multiple blood clots that didn’t respond to standard anti-clotting therapy in a woman with paroxysmal nocturnal hemoglobinuria (PNH), a case study shows. “This report presents a case of myocardial and renal infarction [heart and kidney tissue death due to blood supply obstruction]…
England’s National Institute for Health and Care Excellence (NICE) has recommended that the oral treatment Voydeya (danicopan) be covered by the country’s National Health Service (NHS) and provided at low or no cost to certain adults with paroxysmal nocturnal hemoglobinuria (PNH). The recommendation covers Voydeya’s use as an…
The cases of three people with paroxysmal nocturnal hemoglobinuria (PNH) who experienced serious complications after switching from an experimental medicine to the approved PNH treatment Ultomiris (ravulizumab) highlight the potential for serious risks when switching from a PNH treatment targeting earlier steps of complement activation to one targeting…
Treatment with Empaveli (pegcetacoplan), approved for adults with paroxysmal nocturnal hemoglobinuria (PNH), may lead to clinically meaningful improvements in blood parameters and fatigue among those with impaired bone marrow function. That’s according to a new analysis of data from two Phase 3 clinical trials — PEGASUS (NCT03500549) and…
In rare cases, acute kidney injury may be sign of paroxysmal nocturnal hemoglobinuria (PNH), as described in a recent case report. “Although renal dysfunction in PNH is rare and as renal dysfunction is a dreadful complication, timely intervention is life-saving for the patient who requires hemodialysis,” the scientists wrote.
Treatment with Ultomiris (ravulizumab) enabled paroxysmal nocturnal hemoglobinuria (PNH) patients to achieve the same level of physical and mental health, daily activity, and sleep duration displayed by healthy individuals from the U.S. general population, according to the findings of an observational study. In the study, people with PNH…
A young woman with paroxysmal nocturnal hemoglobinuria (PNH) developed neutropenic enterocolitis (NEC), a severe and life-threatening complication, after receiving treatment with Soliris (eculizumab), a case report indicates. NEC is marked by severe inflammation of the large intestine, which may be associated with infection, due to a markedly low…
Bkemv (eculizumab-aeeb) — a biosimilar of Soliris (eculizumab) that was approved in the U.S. earlier this year as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) — shows equivalent safety and efficacy to its reference drug, according to newly published data from a Phase 3 clinical trial. Results…
The European Commission has approved Roche’s PiaSky (crovalimab) to treat adults and adolescents with paroxysmal nocturnal hemoglobinuria (PNH), ages 12 and older who weigh at least 40 kilograms (88 pounds). PiaSky is the first treatment for PNH in the European Union that’s administered subcutaneously (under the skin) once a…
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