BEIJING and BURLINGTON, Mass., Oct. 16, 2023 /PRNewswire/ --
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 (pCanada, Europe and China. "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." 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. 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. 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. Forward-Looking Statements
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