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Article source: Medical Cube Info
Author: sunshine
On January 10, the CDE official website showed that Beihai Kangcheng's maralixibat oral liquid (maralixibat) is planned to be included in the priority review and approval process due to "new varieties, dosage forms and specifications of children's drugs that meet the physiological characteristics of children" for the treatment of 1 year old.
Cholestatic pruritus in patients with Alagille syndrome (ALGS) and above
.
This is the first and only FDA-approved drug for the rare disease of ALGS
Alagille syndrome is a rare genetic disorder in which bile ducts are abnormally narrowed, deformed, and reduced in number, causing bile to accumulate in the liver, causing progressive liver disease
.
It usually develops in childhood, mutations may involve multiple organ systems, including the liver, heart, kidneys, and central nervous system, and clinical manifestations are pruritus, jaundice, developmental delay, xanthoma, and progressive liver disease
Maximibate is a novel, minimally absorbed, oral investigational drug that inhibits the sodium-dependent bile acid transporter (ASBT), allowing more bile acid to be excreted in the feces, lowering systemic bile acid levels, and potentially reducing bile Acid-mediated liver injury and related effects and complications
.
In September 2021, Mirum announced that the FDA approved maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome 1 year and older
The FDA approval was based primarily on data from a randomized, double-blind, placebo-controlled pivotal Phase IIb ICONIC study (n = 31)
.
Results showed significant improvement in multiple clinical parameters, including pruritus, serum bile acids (sBA), xanthoma, improved quality of life, and growth
On April 29, 2021, Beihai Kangcheng and Mirum reached an exclusive license to develop and commercialize maralixibat in Greater China for ALGS, progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA)
.
In exchange, Mirum is entitled to an upfront payment of $11 million, milestone payments of up to $109 million, and a gradient of double-digit royalties based on sizable net product sales
