The US Food and Drug Administration (FDA) has approved Lynavoy (linerixibat) for the treatment of cholestatic pruritus in adult patients with primary biliary cholangitis (PBC).
PBC is a chronic, progressive autoimmune liver disease in which the bile ducts become inflamed and damaged. This leads to the buildup of bile and causes scarring, cirrhosis, and eventual liver failure. Many people do not have symptoms when they are first diagnosed. Early symptoms may include fatigue, pruritus, and abdominal pain. As the disease progresses, people with PBC may develop weakness, nausea, diarrhea, edema, bone and joint pain, jaundice, dark urine, and xanthomas. It is thought to be caused by a combination of genetic susceptibility and environmental triggers.
Linerixibat is an ileal bile acid transporter (IBAT) inhibitor designed to inhibit bile acid re-uptake, reducing multiple mediators of pruritus in circulation. It is the first medicine approved in the US for this indication.
The approval is based on data from the GLISTEN (NCT04950127) phase 3 double-blind, randomized, placebo-controlled, global clinical trial. The primary and key secondary endpoints of the study were met, demonstrating significant, rapid, and sustained improvements in cholestatic pruritus and itch-related sleep interference versus placebo. The primary endpoint of change from baseline in monthly itch score showed linerixibat significantly improved itch versus placebo over 24 weeks, as measured on a 0-10 numerical rating scale (NRS) for the worst itch.
The safety profile of linerixibat was consistent with previous studies and the mechanism of IBAT inhibition. The most frequently reported adverse events were diarrhea (61%) and abdominal pain (18%), both of which were mostly mild to moderate. Treatment discontinuation due to diarrhea was in 4% of patients versus less than 1% in placebo, and abdominal pain in 4% versus none in placebo.
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To learn more about PBC and other rare autoimmune conditions, visit https://checkrare.com/diseases/autoimmune-autoinflammatory-disorders/