Pharmaceutical Executive December 6, 2023
Pharmaceutical Executive Editorial Staff

Fabhalta (iptacopan) is the first oral monotherapy approved by the FDA for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria.

The FDA has approved Novartis’ Fabhalta (iptacopan) for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), which is the first oral monotherapy to gain approval for this indication.1 The Factor B inhibitor acts proximally in the alternative complement pathway of the immune system, which allows for comprehensive control of red blood cell (RBC) elimination both in and outside of blood vessels.

“An efficacious oral treatment with a demonstrated safety profile could be practice-changing for physicians and help relieve burdens experienced by people with PNH,” Vinod Pullarkat, MD, MRCP, clinical professor, Department of Hematology and Hematopoietic Cell Transplantation,...

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