China-based rare disease specialist CANbridge Pharmaceuticals Inc., (HKG: 1228) has announced receiving market approval from the National Medical Products Administration (NMPA) for its drug maralixibat (CAN108). The drug is approved to treat pruritus due to cholestasis in patients with Alagille Syndrome (ALGS) aged 1 year and above.
Understanding Alagille Syndrome (ALGS) and Its Impact
ALGS is an autosomal dominant multisystem disease that can lead to end-stage liver disease and death. Characterized by underdeveloped bile ducts in children and adolescents, the disease also involves varying degrees of extrahepatic organ involvement, including the cardiovascular system, bones, kidneys, eyes, and appearance. Children with liver involvement often experience chronic cholestasis, leading to severe itching, jaundice, skin xanthoma, and liver enlargement.
Maralixibat’s Mechanism and Global Progress
Originally discovered by US-based Mirum Pharmaceuticals, maralixibat is a barely-absorbed ileal bile acid transporter (IBAT) inhibitor that blocks the enterohepatic circulation of bile acids, reducing bile acid levels in the liver and serum, and resultant liver injury, and relieving pruritus in patients with ALGS suffering from cholestatic pruritus. Mirum won US FDA approval for the drug in September 2021, indicated to treat cholestatic pruritus in patients aged one year and older with ALGS, marketed under the trade name Livmarli. Maralixibat is also under late-stage development for cholestatic liver diseases such as progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA), with indications awarded breakthrough therapy designations (BTDs) and orphan drug designations (ODDs).
CANbridge’s Exclusive License and NMPA Review
CANbridge picked up an exclusive license from Mirum to develop and commercialize maralixibat in Greater China in April 2021, focusing on three rare liver disease indications: ALGS, PFIC, and biliary atresia (BA). The National Medical Products Administration (NMPA) accepted a New Drug Application (NDA) for the drug in ALGS in January last year and awarded it priority review status. The drug was approved as a clinically urgent drug in Boao Lecheng to treat ALGS patients in February 2022.-Fineline Info & Tech
