FDA Approves First Treatment for Rare Pediatric Liver Disease

The US Food and Drug Administration (FDA) has approved maralixibat (LIVMARLI) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age or older.

The minimally absorbed ileal bile acid transporter (IBAT)l, awarded to Mirum Pharmaceuticals, represents the first and only FDA-approved medication for the rare liver disease.

The disease is a rare genetic disorder that causes abnormalities in bile ducts that can result in progressive liver disease with malformed or reduced bile acids in the liver leading to inflammation and liver injury, preventing the liver from working properly. Cholestasis in ALGS is linked to pruritus, 1 of the most common indications for liver transplant in ALGS.

The approval is based on both the ICONIC study and 5 years of data from supportive studies involving 86 patients with ALGS. The results of the ICONIC show a statistically significant reduction in pruritus, 1 of the most common and arduous symptoms, maintained through 4 years.

"Children with Alagille syndrome suffer from cholestatic pruritus, which is serious, unremitting, and debilitating,” Binita M. Kamath, MBBChir, Pediatric Hepatologist, The Hospital for Sick Children, said in a statement. “Their sleep is disrupted, and they endure bleeding and scarring of the skin due to unrelenting scratching.”

ALGS impacts between 2000-2500 children in the US. Mirum has also received a rare pediatric disease priority review voucher in conjunction with the approval.