News

Extensive clotting in the skin’s small blood vessels, known as dermal thrombosis, was identified in a 44-year-old man who suffered multiple strokes and was later diagnosed with paroxysmal nocturnal hemoglobinuria (PNH), according to a case report from India. Researchers noted that “prompt recognition of this unusual PNH-related skin thrombosis…

Empaveli (pegcetacoplan) can ease disease burden for people with paroxysmal nocturnal hemoglobinuria (PNH), reducing signs of red blood cell destruction, lowering the need for blood transfusions, and improving fatigue and quality of life, according to a review of 12 studies conducted after Empaveli’s approval in 2021. “These findings, based…

A stem cell transplant using donor cells from close relatives was curative in people with paroxysmal nocturnal hemoglobinuria (PNH) co-occurring with aplastic anemia, a condition in which the bone marrow fails to produce enough blood cells, according to a study. At a median of nearly five years after the…

Treatment with C5 inhibitors, a class of approved medications for paroxysmal nocturnal hemoglobinuria (PNH) that block activation of the complement cascade — a part of the immune system that is overactive in the disease — was associated with partial recovery of certain immune cell populations, a study in China…

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…

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…