BEIJING and BURLINGTON, Mass., Oct. 9, 2023 /PRNewswire/ -- 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, today 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."
About LIVMARLI
LIVMARLI® (maralixibat) oral solution is a minimally absorbed ileal bile acid transporter (IBAT) inhibitor that blocks the enterohepatic circulation of bile acids, reduces bile acid levels in the liver and serum, reduces the potential for liver injury, and relieves pruritus. LIVMARLI is the first approved drug to treat cholestatic pruritus associated with Alagille syndrome in the US, Europe, and China.
LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases, including BA. LIVMARLI has been granted Breakthrough Therapy designations for ALGS and PFIC type 2, and orphan designations for ALGS, PFIC and BA, by the FDA.
About Alagille Syndrome (ALGS)
Alagille syndrome (ALGS) is an autosomal dominant, multisystem disorder which can lead to end-stage liver disease and death. The birth prevalence of ALGS is between 1/30,000 and 1/50,000 [1], and it has been registered in the National Rare Diseases Registry System of China (NRDRS). ALGS has been included in the "Second National List of Rare Diseases" in China in September 2023.The disease is characterized by a paucity of bile ducts and involvement of extrahepatic organs, such as the kidneys and eyes, as well as bones and the cardiovascular system. All patients experience liver involvement [2, 3], which often manifests as chronic cholestasis (slowed or stalled bile flow), usually in the neonatal period or within the first 3 months after birth. In addition to jaundice, skin xanthoma, and hepatomegaly, patients may also experience severe pruritus [4], which can lead to skin disfigurement, emotional disorders, sleep deprivation, and interruption of school learning, due to scratching in affected children [5]. It seriously affects the growth, development, and quality of life of patients [6] and can lead to liver transplantation [7].
[1]. Kamath et al, JPGN 2018; 67: 148-156 |
[2]. Turnpenny PD, Ellard S. Eur J Hum Genet, 2012;20:251–57. |
[3]. Saleh M, et al. Appl Clin Genet, 2016;9:75–82. |
[4]. Elisofon SA, et al. J Pediatr Gastroenterol Nutr 2010; 51:759–765. |
[5]. Elisofon et al. JPGN. 2010;51: 759-765. |
[6]. Abetz-Webb et al. Hepatology. 2014, 60(4), 526-527. |
[7]. Kamath BM, et al. Hepatol Comms 2020; 4:387–398. |
IMPORTANT SAFETY INFORMATION
LIVMARLI can cause side effects, including:
Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your healthcare provider should do blood tests before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen) or loss of appetite.
Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you.
A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment.
Other common side effects reported during treatment were gastrointestinal bleeding and bone fractures.
About CANbridge Pharmaceuticals Inc.
CANbridge Pharmaceuticals Inc. (HKEX:1228) is a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology. CANbridge has a differentiated drug portfolio, with four approved drugs and a pipeline of 10 assets, targeting prevalent rare disease and rare oncology indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases, as well as glioblastoma multiforme. The CANbridge Next-Generation Innovation and Process Development Facility is developing novel, potentially curative, gene therapies for rare genetic diseases, including Pompe disease, Fabry disease, spinal muscular atrophy (SMA) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies.
For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com.
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