FDA approves treatment for debilitating itch in rare liver disease

The FDA has approved Lynavoy (linerixibat) to treat the itch associated with primary biliary cholangitis (PBC), a rare cholesterol disease that can lead to liver failure. Primary biliary cholangitis is an autoimmune disease that is more prevalent in women. In the United States, approximately 65 out of every 100,000 women have PBC, which usually becomes apparent during midlife, between the ages of 45 and 65. There is currently no cure for this disease, but medication can manage symptoms and slow disease progression.

Cholestatic pruritus is an internal itch that is experienced by up to 89% of people living with PBC. Lynavoy is an IBAT inhibitor, a targeted oral agent to treat cholestatic pruritus. By inhibiting bile acid re-uptake, Lynavoy reduces multiple mediators of pruritus in circulation.

“Cholestatic pruritus has been underestimated and overlooked for far too long, despite its significant impact on people living with PBC. Seeing a treatment specifically developed for chronic itch finally reach patients is a significant step forward and offers hope for those in need,” said Carol Roberts, president of The PBCers Organization, a support group for patients.

Lynavoy was developed by GSK, but Alfasigma, which is located in Italy, recently acquired the worldwide rights to develop, manufacture and commercialize the therapeutic. The companies have not yet provided a launch date or a list price of Lynavoy.
“The FDA’s approval of linerixibat is an important milestone for patients living with cholestatic pruritus in primary biliary cholangitis: it marks the first treatment specifically developed for this rare and debilitating condition. Alfasigma has deep experience in developing and commercializing therapies for serious liver diseases, including PBC, and with our hepatology expertise and global footprint, we are strongly positioned to lead the worldwide commercialization of linerixibat,” Alfasigma CEO Francesco Balestrieri told Managed Healthcare Executive.

The approval of Lynavoy is based on data from the global GLISTEN phase 3 trial, which showed the therapy significantly improved itch compared with a placebo. In the trial, 119 patients were randomized to receive Lynavoy, and 119 patients received a placebo for 24 weeks.

The trial met both primary and key secondary endpoints, demonstrating significant, rapid (at week two) and sustained improvements in cholestatic pruritus and itch-related sleep interference versus placebo. Additionally, more patients in the linerixibat group had clinically meaningful itch improvement with 56% compared with 43% in the placebo group at week 24.

The safety profile of linerixibat was consistent with previous studies, with gastrointestinal side effects more common in the active treatment group. The most common adverse event, diarrhea, was mostly mild in intensity; discontinuation due to diarrhea was 4% in the linerixibat group versus <1% in the placebo group.

The data from the GLISTEN trial were presented in May 2025 at the European Association for the Study of the Liver (EASL) Congress and published in January 2026 in The Lancet Gastroenterology & Hepatology.