Maralixibat treatment leads to height and weight catch-up in patients with Alagille syndrome (ALGS), according to a new study presented at the European Society for Paediatric Gastroenterology Hepatology and Nutrition (ESPGHAN) 54th annual meeting. “Patients with the greatest height disadvantage at baseline had the greatest catch-up in height,” the authors of the study said. Maralixibat…
Maralixibat treatment leads to marked improvements in pruritus and quality of life in patients with Alagille syndrome (ALGS), according to a study published in Hepatology Communications. The finding is based on results from 2 randomized placebo-controlled clinical trials investigating the effect of maralixibat in 57 children with severe cholestasis secondary to ALGS. Maralixibat, marketed under…
Multicenter databases as contextual comparators to current clinical trials may be advantageous in Alagille syndrome (ALGS) and other rare diseases, according to a new study published in Hepatology Communications. The investigation led by Shneider analyzed a prospective longitudinal cohort of children with cholestasis (LOGIC NCT00571272) that met the inclusion and exclusion criteria from a maralixibat…
China’s National Medical Products Administration (NMPA) accepted the New Drug Application (NDA) for CAN108 (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) aged 1 year and above. “That the NMPA has accepted the NDA, which we anticipate will also receive priority review, for CAN108 for [ALGS], our first candidate in…
Children with Alagille syndrome (ALGS) who responded to maralixibat (Livmarli™) treatment showed clinically meaningful improvements in health-related quality of life (HRQoL) when compared to nonresponders. The new results were presented by Mirum Pharmaceuticals, Inc. during the 2021 North American Society for Pediatric Gastroenterology Hepatology and Nutrition Annual Meeting. “These data demonstrate that the significant improvements…
Patients receiving maralixibat treatment for Alagille syndrome (ALGS) may experience gastrointestinal events including abdominal pain and diarrhea, but these symptoms tend to be mild to moderate and transient, and do not lead to discontinuation of treatment. These findings are being presented as a poster at the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition…
Maralixibat (Livmarli™) treatment improves the health-related quality of life of patients with Alagille syndrome, according to a study that will be presented this month at the virtual annual meeting of the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition. The study by Binita Kamath, MD, from the Division of Gastroenterology, Hepatology, and Nutrition at…
A new clinical trial has started recruiting participants to evaluate the safety and tolerability of maralixibat (Livmarli™) in infants with cholestatic liver diseases including Alagille syndrome. The phase 2 open-label study called RISE is sponsored by Mirum Pharmaceuticals and aims to assess whether maralixibat is safe and well-tolerated in children aged less than 1 year…
Two attending physicians commented in The Lancet on the new findings of the phase 2 placebo-controlled, randomized withdrawal period, open-label, extension ICONIC study for Alagille syndrome (ALGS). Maralixibat (Livmarli™), developed by Mirum Pharmaceuticals, is used for patients with cholestatic liver diseases such as ALGS. Alyssa Kriegermeier, MD, and Sarah A. Taylor, MD, from the Ann…
Total bilirubin and serum bile acid levels at week 48 of maralixibat (Livmarli™) treatment, as well as improvement in pruritus and age at which treatment was initiated, are associated with lower long-term rates of clinically important events and liver transplantation. This is according to the results of an analysis examining predictors of long-term event-free survival…
