pharmaphorum December 6, 2023
Novartis’ targeted factor B inhibitor iptacopan has become the first oral monotherapy to be approved by the FDA for rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH).
The drug, which will be sold as Fabhalta, offers an alternative to therapies for PNH like AstraZeneca/Alexion’s complement C5 inhibitors Soliris (eculizumab) and Ultomiris (ravulizumab) and Apellis’ C3 inhibitor Empaveli (pegcetacoplan) that need to be delivered either by infusion or injection. It has been approved with a broad label, covering previously treated and treatment-naïve patients.
Victor Bultó, president of Novartis in the US, said that the approval of Fabhalta “may mean that patients can reset their expectations of living with PNH.”
Analysts at Jefferies have previously said that iptacopan could hit $3.6 billion in...
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