Novartis Receives FDA Approval for Iptacopan as First Oral Monotherapy for PNH

Novartis today announced that the FDA has approved Fabhalta (iptacopan) as the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria (PNH). In the Phase 3 APPLY-PNH trial, iptacopan demonstrated superior hemoglobin response rates compared to anti-C5 therapy, with 82% of patients achieving hemoglobin increase of ≥2 g/dL. The oral administration of iptacopan offers a convenient alternative to intravenous anti-C5 therapies and represents a significant advancement in PNH treatment. This approval provides patients with a new, effective, and convenient treatment option.