Clinical trials play a crucial role in better understanding rare diseases and therapeutic interventions. According to a news release from rare disease company Albireo Pharma, Inc. (“Albireo”), enrollment is now complete for the Phase 3 BOLD clinical trial. During the trial, researchers will be evaluating the safety, efficacy, and tolerability of Bylvay (odevixibat) for people with biliary atresia.
205 pediatric patients have enrolled in nineteen different countries. They will be followed for a period of 24-months. The goal is to measure the impact of Bylvay on liver function and survival in children with biliary atresia who underwent the Kasai procedure prior to turning three months old. Ultimately, the primary efficacy endpoint will explore how many children required a liver transplant prior to two years old.
What is Bylvay?
Bylvay is an orally-administered non-systemic ileal bile acid transport inhibitor (IBATi) that is designed to reduce the accumulation of bile acids in the liver. Taken once per day, Bylvay has the potential to protect liver function and enhance response to the Kasai procedure; ultimately, if successful, this could reduce the need for liver transplants in infants. Bylvay is also approved for the treatment of pruritus (excessive itchiness) in patients aged 3 months+ with progressive familial intrahepatic cholestasis (PFIC), and being explored as a potential therapy for Alagille syndrome. Additionally, it has received Orphan Drug designation in the U.S. for Alagille syndrome, PFIC, primary biliary cholangitis (PBC), and biliary atresia.
Bylvay has been found to be relatively safe and well-tolerated. However, some side effects may occur. These can include nausea and vomiting, diarrhea, fat-soluble vitamin deficiency, and abdominal pain.
Biliary Atresia: A Brief Overview
Biliary atresia is a rare liver and bile duct disease in infants. In biliary atresia, the bile ducts within and outside of the liver are destroyed, scarred, or blocked. Normally, bile moves from the liver to the gallbladder. However, because of the scarring or damage caused by biliary atresia, bile begins to accumulate in the liver, causing damage, scarring, and loss of function. Biliary atresia is slightly more common in females than males. While the exact cause is unknown, doctors believe the bile ducts become damaged in the womb or shortly following birth. Symptoms typically appear within the first month of life and can (but do not always) include:
- Pale stool and dark urine
- Jaundice (yellowing of the skin, eyes, and mucous membranes)
- Abdominal distention/swelling
- Failure to thrive
- An abnormally enlarged liver
- High blood pressure
- Pruritus (intense itchiness)
- Liver scarring or failure
- Heart and kidney abnormalities
- An absent spleen or extra spleens
