Novartis gets FDA approval of closely watched rare disease drug

BioPharma Dive December 6, 2023
Ned Pagliarulo

Now cleared for paroxysmal nocturnal hemoglobinuria, Fabhalta is also being developed for a range of other so-called complement diseases.

Dive Brief:

The Food and Drug Administration has approved a new Novartis drug for adults with a rare and serious blood disorder known as paroxysmal nocturnal hemoglobinuria, or PNH.
The drug, which Novartis will sell as Fabhalta, was discovered and developed by the Swiss pharmaceutical company for diseases like PNH that are driven by dysregulation of the complement system, part of the body’s immune defenses.
An oral treatment, Fabhalta helped improve hemoglobin levels in clinical studies of people with PNH, which is characterized by the premature destruction of red blood cells by the complement system. This “hemolysis” causes...

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2023-12-06T18:01:08-05:00