FDA Approves Therapy for Rare Liver Disease in Children

The FDA has approved Mirum Pharmaceuticals’ Livmarli (maralixibat) oral solution to treat cholestatic pruritus in Alagille syndrome (ALGS) in children one year of age and older.

Livmarli, a minimally absorbed ileal bile acid transporter (IBAT) inhibitor, is the first FDA-approved medication for the rare liver disease, which affects 2,000 to 2,500 children in the United States, company officials said in a news release. The medication is available immediately.

ALGS is a rare, genetic disorder caused by abnormalities in bile ducts that can lead to progressive liver disease. Malformed or reduced bile ducts cause cholestasis, the accumulation of bile acids in the liver, which leads to inflammation and liver injury, and prevents the liver from working properly.

Binita M. Kamath, MBBChir.

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

Livmarli’s approval is based on the pivotal ICONIC study, as well as five years of data from supportive studies. Murium’s ICONIC study demonstrated statistically significant reductions in pruritus, which was maintained for four years, Mirium said.

Livmarli will be accessible to patients through Mirum Access Plus (MAP), the company’s patient support services program and single-source specialty pharmacy. “The MAP program has fully dedicated and experienced coordinators who will work with healthcare providers and families to provide insurance coverage and access support, as well as help with financial support options for Livmarli,” the company said.

Mirum Pharmaceuticals projects that the market for Livmarli is worth around $500 million.