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…
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…
Close monitoring and Empaveli (pegcetacoplan) dose adjustments before and after surgery effectively prevented episodes of breakthrough hemolysis,in a 67-year-old man with paroxysmal nocturnal hemoglobinuria (PNH), according to a case report in Spain. Hemolysis is a hallmark symptom of PNH marked by the breakdown of red blood cells, which…
Clinical site activation is now ongoing for a Phase 3 program that will evaluate zaltenibart (OMS906) as a treatment for people with paroxysmal nocturnal hemoglobinuria (PNH), its developer Omeros has announced. A total of 120 sites across 30 countries were chosen for clinical trial participation, according to a…
An unusual meningococcal infection during treatment with Empaveli (pegcetacoplan) was reported in a 16-year-old girl with paroxysmal nocturnal hemoglobinuria (PNH), according to a case report from Finland. Meningococcal infections are a known risk for patients receiving complement inhibitors, such as Empaveli. As such, patients typically are vaccinated before…
Note: This story was updated Dec. 6, 2024, to correct that Voydeya is approved as an add-on treatment to standard PNH therapies to help control extravascular hemolysis. The headline was changed to clarify that these were the results of a review study. Voydeya (danicopan) — whether given as a…
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…
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 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…
A 43-year-old woman with paroxysmal nocturnal hemoglobinuria (PNH) who experienced extravascular hemolysis — which occurs when red blood cells are destroyed or broken down outside of the blood vessels — despite previous treatment saw her condition significantly improve after initiating treatment with Fabhalta (iptacopan), according to a case…
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