Novartis Nabs FDA Nod for Rare Disease Drug Rival to AstraZeneca Meds

MedCity News December 6, 2023
Frank Vinluan

FDA approval of Novartis’s Fabhalta makes the drug the first approved oral therapy for rare blood disorder paroxysmal nocturnal hemoglobinuria. With clinical data showing superiority versus two infused AstraZeneca drugs, Novartis’s pill is well-positioned to take market share from those blockbuster products.

A rare blood disorder treatable with infused medications now has its first oral drug alternative. FDA approval of the new drug from Novartis positions the pill to compete directly against two blockbuster AstraZeneca products.

The rare disease, paroxysmal nocturnal hemoglobinuria (PNH), leads to the destruction of red blood cells by the complement system, a part of the immune system. The Wednesday approval of the Novartis drug, iptacopan, covers adults regardless of whether they have previously been treated with...

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