Novartis

Novartis announced new Phase II data for iptacopan (LNP023), an investigational oral treatment for paroxysmal nocturnal hemoglobinuria (PNH), presented at the 26th Annual Congress of the European Hematology Association (EHA).

The U.S. Food and Drug Administration approved the expanded use of Alexion Pharmaceuticals Inc.’s Ultomiris (ravulizumab-cwvz) to include children (1 month of age and older) and adolescents with paroxysmal nocturnal hemoglobinuria (PNH).

Novartis announced Phase II primary endpoint data showing the investigational, first-in-class, oral, targeted factor B inhibitor iptacopan reduced protein in the urine – an increasingly recognized surrogate marker correlating with progression to kidney failure – and showed promise in stabilizing kidney function in patients with IgA nephropathy (IgAN).

The U.S. Food and Drug Administration approved Apellis Pharmaceuticals’ Empaveli as the first targeted C3 therapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).

Novartis

Novartis received the U.S. Food and Drug Administration’s Breakthrough Therapy tag for the company’s experimental medicine iptacopan, which the Swiss drugmaker hopes wins approval to treat the rare blood disorder paroxysmal nocturnal hemoglobinuria.

The U.S. FDA gave Alexion Pharmaceuticals a thumbs-up for Soliris (eculizumab) to treat neuromyelitis optica spectrum disorder in adults that express a specific biomarker.

Alexion Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration accepted for review the company’s Biologics License Application for approval of ALXN1210, an investigational long-acting C5 complement inhibitor, for the treatment of patients with paroxysmal nocturnal hemoglobinuria.