Iptacopan FDA Submission Coming Soon: Novartis

With positive results from the Phase 3 APPOINT-PNH trial showing that the investigational oral monotherapy iptacopan improves hemoglobin to near-normal levels in treatment-naïve paroxysmal nocturnal hemoglobinuria (PNH) patients, Novartis says it is planning global regulatory submissions, including FDA, in the first half of this year. The company says the drug met its primary endpoint and demonstrated clinically meaningful benefits across secondary endpoints.

In addition to the regulatory submissions, company officials say, they are continuing to progress iptacopan studies for a range of other complement-mediated diseases.

Iptacopan is described as an investigational first-in-class orally administered targeted factor B inhibitor of the alternative complement pathway that acts upstream of the C5 terminal pathway, preventing intravascular and extravascular hemolysis in PNH. It has received FDA breakthrough therapy and orphan drug designations for PNH.