Iptacopan for Rare Diseases Receives Dual FDA Designations

Novartis’ iptacopan (LNP023) has received a Breakthrough Therapy designation based on positive interim results from two phase 2 trials for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare life-threatening blood disorder.

The company also announced that the investigational oral drug has received a Rare Pediatric Disease designation from the agency for the treatment of C3 glomerulopathy (C3G), an ultra-rare kidney disease.

Novartis believes iptacopan has the potential to become the first complement pathway inhibitor to slow disease progression in several complement-driven diseases and is planning phase 3 studies for several indications.

Iptacopan previously received Orphan Drug designations from the FDA and European Medicines Agency for the treatment of PNH and C3G.