Novartis has received FDA approval for Fabhalta® (iptacopan), making it the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria (PNH). Fabhalta, a Factor B inhibitor, controls red blood cell (RBC) destruction within and outside blood vessels. Clinical trials demonstrated increased hemoglobin levels in most patients, and in APPLY-PNH, almost all Fabhalta-treated patients did not require blood transfusions. The FDA approval is based on the Phase III APPLY-PNH trial, showing superiority over anti-C5 treatments in hemoglobin improvement and transfusion avoidance. The safety profile revealed common adverse reactions such as headache, nasopharyngitis, diarrhea, and abdominal pain.

PNH is a rare and serious blood disorder characterized by complement-mediated hemolysis, bone marrow failure, and thrombosis. Existing treatments may leave some PNH symptoms uncontrolled, leading to persistent anemia and the need for blood transfusions.

Fabhalta is expected to be available in the U.S. starting December, with additional regulatory filings and reviews underway globally. Novartis aims to explore Fabhalta's potential in other complement-mediated diseases. The FDA approval is a significant development for people with PNH, providing a new and effective oral treatment option that may positively impact their quality of life.

Source: prnewswire.com