Unfortunately, clinical studies do not always work out the way they’re expected to. Biopharmaceutical company Mirum Pharmaceuticals, Inc. (“Mirum”) learned this after the Phase 2 EMBARK trial failed to meet its primary endpoint—mean bilirubin change from baseline by Week 26 of the study—or key secondary endpoints. According to a December 2023 news release from the company, the trial sought to understand whether LIVMARLI (maralixibat) could be used as an adjuvant treatment following the Kasai procedure, an operation used to treat a rare liver and bile duct disease called biliary atresia. During the course of the trial, participants were given either LIVMARLI or a placebo.
LIVMARLI is a prescription medication and oral solution that is currently approved to treat cholestatic pruritus in patients with Alagille syndrome ages three months or older. Mirum is now evaluating the drug for biliary atresia and has submitted LIVMARLI for approval in the indication of progressive familial intrahepatic cholestasis. Learn more about LIVMARLI.
What to Know about Biliary Atresia
As described above, biliary atresia is a rare condition in which bile ducts within and outside of the liver become damaged, destroyed, blocked, or scarred. Our bile ducts normally transport bile from the liver to the gallbladder and small intestine. When the bile ducts aren’t working properly, as in the case of biliary atresia, bile builds up in the liver and damages it. This can lead to a number of health issues such as liver cirrhosis and loss of liver function.
The exact cause of biliary atresia remains unknown. However, the current hypothesis stands that the bile ducts become damaged either in the womb or shortly after birth. Biliary atresia is treated using the Kasai procedure and, in many cases, an eventual liver transplant.
Symptoms of biliary atresia often appear within the first month of life. These symptoms may include intense itchiness (known as pruritus), pale stool and dark urine, yellowing of the skin and eyes (known as jaundice), failure to thrive, an abnormally enlarged liver, an absent or extra spleen, heart and kidney abnormalities, high blood pressure, and a swollen abdomen.
