GC said it has applied for product approval for Maralixibat, an Alagille syndrome (ALGS) treatment, to the Food and Drug Safety Ministry.

ALGS is a rare genetic disease in children in which the hepatic biliary tract decreases and bile stagnates, and the only available treatment is currently liver transplantation.

GC signed a technology transfer contract with Mirum Pharmaceuticals, a U.S. pharmaceutical company, in July last year for exclusive development and commercialization of Maralixibat in Korea.

The drug received a breakthrough therapy designation from the U.S. Food and Drug Administration in 2019 and received approval for use in treating cholestatic pruritus in patients with ALGS in September last year.

Mirum Pharmaceuticals also applied for approving its treatment to the European Medicines Agency and is undergoing approval procedures and clinical trials to expand the indication of the drug to treat progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA) in the U.S. and Europe.

Starting with the ALGS indication approval for Maralixibat, GC plans to pursue product approval for all three indications in Korea.

"Maralixibat is the only new drug in Korea's rare liver disease market," a company official said. "We expect that this drug to be a new hope for patients with such rare diseases."