Paroxysmal Nocturnal Hemoglobinuria

Data confirm the safety and efficacy of ravulizumab among patients with paroxysmal nocturnal hemoglobinuria previously treated with eculizumab, according to results of the REACTION study presented at ASH Annual Meeting and Exposition.

Sobi and Apellis Pharmaceuticals Inc. announced results from the phase 3 Valiant study showing the drug pegcetacoplan reduced proteinuria and stabilized eGFR compared to placebo in patients with glomerulopathy or glomerulonephritis.

LONG BEACH, Calif. — Iptacopan may be superior to placebo in reducing proteinuria at 9 months, with early onset and effect consistency, according to data presented at the National Kidney Foundation Spring Clinical Meetings, here.

The FDA approved iptacopan for treatment of adults with paroxysmal nocturnal hemoglobinuria.

Crovalimab provided disease control and proved noninferior to a current standard-of-care treatment for certain patients with paroxysmal nocturnal hemoglobinuria, according to the agent’s manufacturer.

NEW ORLEANS — Patients with paroxysmal nocturnal hemoglobinuria who received iptacopan vs. standard therapy had clinically meaningful and significant improvement in symptoms, results of the phase 3 APPLY-PNH trial showed.

Peter Hillmen, MD, PhD, and John Atkinson, MD, will receive the Ernest Beutler Lecture and Prize at this year’s ASH Annual Meeting & Exposition.

The FDA approved ravulizumab-cwvz injection to treat children aged 1 month and older with paroxysmal nocturnal hemoglobinuria.

The FDA approved pegcetacoplan for treatment of certain adults with paroxysmal nocturnal hemoglobinuria, according to the agent’s manufacturer.

Pegcetacoplan led to greater improvements in hemoglobin compared with eculizumab among patients with paroxysmal nocturnal hemoglobinuria, according to results of the phase 3 PEGASUS study published in The New England Journal of Medicine.