The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the approval of Novartis’s (NYSE: NVS) Factor B inhibitor Fabhalta (iptacopan) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adults with hemolytic anemia. The European Commission (EC) is anticipated to make a decision within the next two months.
The recommendation follows the review of Phase III data by the EMA, which demonstrated that 92.2% of complement inhibitor-naive patients experienced a sustained improvement in hemoglobin levels without the need for transfusions. Furthermore, the transition from an anti-C5 treatment to Fabhalta increased the rate of transfusion independence from 2.0% to 82.3%.
As an oral medication, Fabhalta has the potential to revolutionize the treatment of PNH, where current injectable anti-C5 therapies result in 23% to 39% of patients remaining dependent on blood transfusions. Fabhalta has shown a transfusion avoidance rate of 94.8%, positioning it as a significant advancement in the management of PNH.- Flcube.com
