Treatment with Ultomiris (ravulizumab) safely helps maintain or achieve disease control in people with paroxysmal nocturnal hemoglobinuria (PNH), according to a real-world study involving more than 200 adults. Among that group, people who switched from the older therapy, Soliris (eculizumab), to…
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The first patient has been dosed in Argo Biopharmaceutical’s Phase 2 clinical trial of BW-40202 for paroxysmal nocturnal hemoglobinuria (PNH), one of two new Phase 2 studies testing the investigational therapy. The Phase 2 studies follow a Phase 1 trial (NCT06917482) initiated last year to assess BW-40202’s…
Doctors in China diagnosed a man with paroxysmal nocturnal hemoglobinuria (PNH) after a biopsy and specialized MRI, performed to assess kidney problems, yielded clues about ongoing red blood cell breakdown. The tests identified iron buildup in the man’s kidneys (renal hemosiderosis). The findings raised suspicion of ongoing red blood…
Fabhalta (iptacopan) appears to outperform Empaveli (pegcetacoplan) in raising hemoglobin levels and reducing the need for blood transfusions in adults with paroxysmal nocturnal hemoglobinuria (PNH) who continue to have symptoms despite previous treatment. Those are the findings of a new study that indirectly compared the two approved…
Biogen has entered into an agreement to acquire Apellis Pharmaceuticals, adding Empaveli (pegcetacoplan), marketed in Europe as Aspaveli, to its portfolio for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and two other rare diseases involving abnormal complement activation, which is part of the immune system. For Biogen,…
The approved dosing regimens of Voydeya (danicopan) are supported for adults with paroxysmal nocturnal hemoglobinuria (PNH) receiving standard therapies and generally do not appear to require adjustment based on patient characteristics or food intake, according to a new analysis of clinical trial data. Voydeya is an oral medication…
Blood markers designed to measure activation of the complement system, a part of the immune system involved in paroxysmal nocturnal hemoglobinuria (PNH), may not reliably reflect disease activity or red blood cell destruction in patients receiving complement-blocking therapies, a new study suggests. The researchers found that although these complement…
The first clinical report of a patient with paroxysmal nocturnal hemoglobinuria (PNH) switching from PiaSky (crovalimab-akkz) to Fabhalta (iptacopan) was reported recently. The patient in the report was a 72-year-old man who continued to experience PNH symptoms after an infection triggered…
Antibody-based therapy ruxoprubart (formerly NM8074) safely prevented blood cell destruction, eased anemia, and eliminated the need for blood transfusions when given as a standalone treatment in adults with paroxysmal nocturnal hemoglobinuria (PNH). That’s according to results from a Phase 2 trial (NCT05646524) announced by its developer,…
Use of the approved therapy Ultomiris (ravulizumab) keeps hemolysis, or the destruction of red blood cells, under control, reduces the need for transfusions, and is generally well tolerated for people with paroxysmal nocturnal hemoglobinuria (PNH), a real-world study in Italy has found. According to the researchers, the benefits…
Recent Posts
- Real-world data support Ultomiris for first-line PNH treatment
- I’m finding my rhythm again thanks to a positive turn in my PNH journey
- First patients dosed in Phase 2 trials of Argo’s complement therapy BW-40202
- Iron in kidneys leads doctors to diagnose man’s PNH: Case report
- I’m navigating a new chapter with PNH, one between fear and hope