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Use of the approved injection therapy Empaveli (pegcetacoplan) appears to be effective and generally safe for people with paroxysmal nocturnal hemoglobinuria (PNH) — especially for patients who, despite treatments, have ongoing destruction of red blood cells, known as hemolysis. Those are the findings of a new analysis from…

Treatment with Soliris (eculizumab) appears to help reduce the risk of potentially life-threatening thromboembolic events (TEs), complications caused by clots that disrupt circulation, in people with paroxysmal nocturnal hemoglobinuria (PNH), but it does not eliminate that risk, according to a post-marketing surveillance study from Japan. Researchers found that…

Most adults with paroxysmal nocturnal hemoglobinuria (PNH) who switched from standard C5 inhibitors to Fabhalta (iptacopan) saw their hemoglobin levels rise, remained free from blood transfusions, and reported less fatigue after six months of treatment, according to Phase 3 trial results. The open-label Phase 3b APPULSE-PNH study (NCT05630001) was designed…

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…