The FDA approved maralixibat (Livmarli) as the first authorized treatment for cholestatic pruritus in patients with Alagille syndrome (ALGS), a rare liver disease.
ALGS is a rare genetic disorder in which bile builds up in the liver, leading to inflammation, injury, and, eventually, progressive liver disease. And one of the more troublesome side effects of the condition is cholestatic pruritus, which can range from mild to severe. Maralixibat, a once-daily, minimally absorbed ileal bile acid transporter inhibitor, is approved to treat the side effect in ALGS patients one year of age and older.
For children dealing with ALGS, cholestatic pruritus can be “serious, unremitting, and debilitating. Their sleep is disrupted, and they endure bleeding and scarring of the skin due to unrelenting scratching,” said Binita M. Kamath, MBBChir, Pediatric Hepatologist, The Hospital for Sick Children (SickKids), Toronto, Ontario, Canada, said in a company press release. “There have been no approved treatments to date for cholestatic pruritus in Alagille syndrome, and many children ultimately require major surgical interventions such as liver transplantation for refractory pruritus. The approval of [maralixibat] signifies a meaningful shift in the treatment paradigm for Alagille syndrome and provides hope for the many families who have lived with persistent itch for far too long.”
This approval was based on results from the ICONIC data, as well as five years of data from supportive studies among 86 patients with ALGS. “Data from ICONIC demonstrated statistically significant reductions in pruritus, one of the most common and arduous symptoms associated with the disease, which was maintained through four years,” the drug’s manufacturer explained.
The manufacturer warned that maralixibat can cause serious side effects, including changes in liver tests, diarrhea, stomach pain, vomiting, fat soluble vitamin (FSV) deficiency, bone fractures, and gastrointestinal bleeding.
Maralixibat is manufactured by Mirum Pharmaceuticals, Inc.
John McKenna, Associate Editor, BreakingMED™
Cat ID: 192
Topic ID: 86,192,730,111,192,725,925
