CANbridge Announces NDA Acceptance of CAN108 (邁芮倍®/LIVMARLI®) for Cholestatic Pruritus in Progressive Familial Intrahepatic Cholestasis (PFIC) by China's National Medical Products Administration

BEIJING and BURLINGTON, Mass., Oct. 16, 2023 /PRNewswire/ -- CANbridge Pharmaceuticals Inc. (HKEX:1228), a global biopharmaceutical company, with a foundation in China,  committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, announced today that the National Medical Products Administration of China (NMPA) has accepted the New Drug Application (NDA) for CAN108 (Maralixibat Chloride Oral Solution /迈芮倍®/LIVMARLI®) for the treatment of cholestatic pruritus in patients two months of age and older with progressive familial intrahepatic cholestasis (PFIC). PFIC is a rare genetic disorder that causes progressive liver disease typically leading to liver failure. PFIC has been included in the "First National List of Rare Diseases" in China since May 2018. The application has been granted priority review.

The submission is based on data from the MARCH PFIC Phase 3 study of LIVMARLI. MARCH PFIC is the largest randomized trial conducted in PFIC, with 93 patients across a range of genetic PFIC subtypes, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6 and unidentified mutational status. In the study, LIVMARLI-treated patients had statistically significant improvements in pruritus (p< 0.0001), serum bile acids (p<0.0001), bilirubin (p=0.0471), and growth as measured by weight z-score (p=0.0391), in the cohort evaluating combined genetic subtypes.

LIVMARLI is the first approved drug to treat cholestatic pruritus associated with Alagille syndrome in the US, Canada, Europe and China. 

CANbridge and Mirum Pharmaceuticals, Inc. (Mirum) signed an exclusive license agreement for the development, manufacturing and commercialization of LIVMARLI in Greater China in 2021. Mirum has also submitted a supplemental NDA to the U.S. Food and Drug Administration (FDA) for the treatment of cholestatic pruritus in patients three months of age and older with PFIC, as well as a marketing authorization variation to the European Medicines Agency (EMA) for the treatment of PFIC. Under the terms of the agreement, CANbridge has the right to develop manufacture and commercialize LIVMARLI for three indications: Alagille syndrome (ALGS), PFIC and biliary atresia (BA), along with select other indications, in Greater China. LIVMARLI is currently being evaluated in an ongoing Global Phase 2 study in China known as EMBARK for the treatment of BA.

"The acceptance of the NDA for LIVMARLI in PFIC by the NMPA is another step towards our goal of bringing treatments to patients in China who suffer from rare diseases," said James Xue, Ph.D., CANbridge Founder, Chairman and CEO. "With the approval of Livmarli for the treatment of patients with ALGS in mainland China, Hong Kong and Taiwan already received earlier this year, PFIC represents the opportunity for CANbridge to expand the label and maximize the potential of this product.  We eagerly anticipate the formal approval of this indication in China, where it holds the potential to provide much-needed relief to patients and their families."

About PFIC

Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive liver disease typically leading to liver failure. In people with PFIC, liver cells are less able to secrete bile. The resulting buildup of bile causes liver disease in affected individuals. Signs and symptoms of PFIC typically begin in infancy. Patients experience severe itching, jaundice, failure to grow at the expected rate (failure to thrive), and an increasing inability of the liver to function (liver failure). The disease is estimated to affect one in every 50,000 to 100,000 births in the United States and Europe.  Six types of PFIC have been genetically identified, all of which are similarly characterized by impaired bile flow and progressive liver disease.

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 resultant liver injury and relieves pruritus. LIVMARLI is the first approved drug to treat cholestatic pruritus associated with Alagille syndrome in the US, Canada, Europe and China. 

LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including biliary atresia.  LIVMARLI has been granted Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia by the FDA.

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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SOURCE CANbridge Pharmaceuticals, Inc.