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Novartis’ Fabhalta approved by FDA as first oral monotherapy for rare blood disease PNH

December 6, 2023


PNH patients have an acquired mutation that causes them to produce red blood cells susceptible to premature destruction by the complement system, which can cause anaemia, thrombosis, fatigue and other symptoms that can impact quality of life.

The disease has a significant unmet need, Novartis reports, with a large proportion of patients on anti-C5 treatment remaining anaemic and dependent on blood transfusions.

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