Novartis' orphan drug wins approval to treat paroxysmal nocturnal hemoglobinuria

The Ministry of Food and Drug Safety (MFDS) said Thursday that it has approved Fabhalta Capsules (iptacopan), a treatment for paroxysmal nocturnal hemoglobinuria (PNH) imported by Novartis Korea.

Paroxysmal nocturnal hemoglobinuria is a rare disease caused by a hematopoietic stem cell gene mutation that makes red blood cells vulnerable to the complement response and causes hemolysis in and around blood vessels, resulting in symptoms such as persistent anemia, fatigue, and venous blood clots.

Fabhalta Capsule specifically binds to complement factor B. It inhibits the production of the C5 convertase complex by inhibiting the C3 convertase of the alternative complement pathway.

Fabhalta Capsule has improved efficacy over existing therapies by inhibiting extravasation hemolysis that can occur with existing therapies. The ministry said Fabhalta Capsule is expected to help expand the treatment options for adult patients with paroxysmal nocturnal hemoglobinuria.

In June last year, the MFDS designated Fabhalta Capsule as the fifth Global Innovative Product Fast Track (GIFT) and expedited its review to supply it to the medical field quickly.

“We will continue to do our best to ensure that therapeutic products with sufficiently confirmed safety and effectiveness are quickly reviewed and approved based on regulatory science expertise to help expand patient treatment opportunities,” the ministry said.