The FDA on Dec. 6 approved Novartis’ Fabhalta (iptacopan) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. This is the first FDA approval for the factor B inhibitor, which also has a pending application for immunoglobin A nephropathy. Fabhalta will also be the first oral monotherapy approved to treat PNH, which is typically treated with C5 inhibitors such as Soliris and Ultomiris, both from AstraZeneca. The drugs are administered as injectables or infusions, vs. Fabhalta’s oral route. For the treatment of PNH, Soliris and Ultomiris currently hold covered or better status for 86% of all insured lives under the medical benefit.
SOURCE: MMIT Analytics, as of 12/11/23