09 October 2023 | Monday | News
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CANbridge Pharmaceuticals Inc. (1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare diseases and oncology, announced the marketing approval of CAN108 (Maralixibat Chloride Oral Solution /LIVMARLI®) by the Taiwan Food and Drug Administration (TFDA).
LIVMARLI is a minimally absorbed ileal bile acid transporter (IBAT) inhibitor, and the first and only treatment approved medication in Taiwan for the treatment of cholestatic pruritus (itching caused by slowed or stalled bile flow) in patients with Alagille syndrome (ALGS) aged one year or older. ALGS is a rare genetic disorder that can lead to end-stage liver disease and death. ALGS is characterized by paucity of the bile ducts, which causes cholestasis, and involvement of extrahepatic organs, such as the kidneys, eyes, as well as bones and the cardiovascular system. Cholestatic pruritus is the most burdensome symptom in ALGS, greatly reducing quality of life. LIVMARLI has recently been approved in Hong Kong, mainland China and Canada. It has also been approved in the United States for patients with ALGS aged three months and older, and in Europe for patients with ALGS aged two months and older. This expansion reflects CANbridge's commitment to providing access to advanced therapies for rare diseases on a global scale.
The approval of LIVMARLI is based on data from Mirum Pharmaceuticals' pivotal ICONIC study and RISE infant safety study, in addition to years of data collected from the program in patients with ALGS. The ICONIC study demonstrated statistically significant and clinically meaningful reductions in pruritus compared to placebo, as well as significant reductions in serum bile acids, both of which were durably maintained over several years of treatment. Interim data from the RISE study supports the indication in infant group at age of less than 12 months.
CANbridge holds an exclusive license with Mirum Pharmaceuticals, Inc. for the development, commercialization, and manufacturing, under certain conditions, of LIVMARLI in Greater China for three rare liver disease indications: Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA), along with other selected indications. LIVMARLI is currently being evaluated in an ongoing Global Phase 2 study known as EMBARK for the treatment of BA, which completed patient enrolment in China in May 2023.
"We are thrilled by the approval of LIVMARLI in Taiwan, following the approvals in mainland China and Hong Kong over the span of six months, signifying a major stride forward in our mission to enhance the lives of rare disease patients," said James Xue, Ph.D., Founder, Chairman and CEO of CANbridge Pharmaceuticals Inc. "Bringing this important therapy to Taiwan not only underscores our commitment to innovation but also reflects our dedication to making advanced treatments accessible to patients in need globally. The availability of LIVMARLI in Taiwan represents a significant turning point in addressing the unique challenges faced by individuals affected by ALGS. We extend our sincere appreciation to our partner, Mirum, for their unwavering support in this remarkable journey."