GC Green Cross Applies for Marketing Authorization of Pediatric Rare Epilepsy Treatment 'Maralixibat'

[Asia Economy Reporter Lee Gwan-joo] GC Green Cross announced on the 2nd that it has applied for marketing approval of the pediatric rare liver disease drug ‘Maralixibat’ to the Ministry of Food and Drug Safety.

Maralixibat is a treatment for Alagille Syndrome (ALGS), a rare pediatric genetic disorder characterized by decreased bile ducts and bile stasis in the liver. Currently, liver transplantation is the only treatment for ALGS.

In July last year, GC Green Cross signed a license agreement with Mirum Pharmaceuticals in the United States for exclusive domestic development and commercialization of Maralixibat. Maralixibat was designated as a Breakthrough Therapy by the U.S. Food and Drug Administration (FDA) in 2019 and received FDA marketing approval last September as a treatment for cholestatic pruritus in ALGS.

Mirum has also submitted a marketing authorization application to the European Medicines Agency for the same indication. Additionally, clinical trials and approval procedures for other indications such as Progressive Familial Intrahepatic Cholestasis (PFIC) and Biliary Atresia (BA) are underway in both the U.S. and Europe.

GC Green Cross plans to proceed with domestic marketing approval procedures for a total of three indications, starting with ALGS. Maralixibat is expected to become a new hope for patients suffering from rare liver diseases as the only new drug in the domestic market.

Orphan drugs refer to treatments used for diseases with fewer than 20,000 patients domestically, for which appropriate treatment methods and drugs have not been developed, or drugs that show significantly improved safety or efficacy compared to existing alternatives. When designated as an orphan drug, benefits such as exemption from Drug Master File (DMF) review and bridging studies can accelerate the product launch timeline.

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