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…
Increasing dosing of Empaveli (pegcetacoplan) followed by co-administering Soliris (eculizumab) effectively managed acute red blood cell destruction in a woman with paroxysmal nocturnal hemoglobinuria (PNH), a study shows. The effort underscores how acute red blood cell destruction “requires prompt diagnosis and treatment to avoid potentially…
Health Canada has approved the oral therapy Voydeya (danicopan) as an add-on to Ultomiris (ravulizumab) or Soliris (eculizumab) in adults with paroxysmal nocturnal hemoglobinuria (PNH) who have residual anemia due to blood cell destruction taking place outside blood vessels. The announcement came just a few months…
The specialty pharmacy Onco360 was selected to supply PiaSky (crovalimab-akkz), an antibody-based medication that works to reduce red blood cell destruction, to teenagers and adults with paroxysmal nocturnal hemoglobinuria (PNH). Marketed by Genentech (a Roche subsidiary) in the U.S., the treatment was recently approved by…
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