The results from the phase 3 APPEAR-C3G study showed that Fabhalta (iptacopan) met its primary objective of an improvement of protein in the urine (proteinuria) compared to placebo at six months, setting up filings for regulatory approval.
Last week, targeted factor B inhibitor Fabhalta (iptacopan) was cleared by the FDA as an oral 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.
