FDA Approves Novartis Iptacopan for rare blood disease PNH Read More

FDA has approved Novartis’ Iptacopan (Fabhalta) for treatment of rare blood disease, Paroxysmal Nocturnal Hemoglobinuria (PNH). It is the first oral monotherapy for the treatment of PNH.

Current treatments for PNH are AstraZeneca’s Soliris and Ultomiris, both are infusions, and Fabhalta is an oral pill taken twice a day.

Regulatory filings and reviews for Fabhalta in PNH are currently underway around the world. Fabhalta is also currently in development for a range of complement-mediated diseases and has potential to become a block buster drug.

Fabhalta comes with a boxed warning as per FDA label and will be available only through a restricted Risk Evaluation and Mitigation Strategy (REMS) program.

Novartis Announcement

FDA Label