Phase 3 Trials for an Investigational Progressive Familial Intrahepatic Cholestasis Drug are Gearing Up

According to a story from Financial Buzz, the pharmaceutical company Albireo Pharma recently announced that the first patient has been enrolled in its Phase 3 trial testing its experimental candidate A4250. The drug is in development for the treatment of progressive familial intrahepatic cholestasis (PFIC). Albireo Pharma specializes in the development of drugs for pediatric liver diseases.
PFIC is a type of cholestasis, a disease in which the flow of bile from the liver is restricted. While cholestasis can be caused by a variety of different factors, PFIC is a genetically linked type in which the biliary epithelial transporters are defective. The condition first occurs early in life, and leads to failure to thrive in infancy. Other symptoms of PFIC include intense itching and jaundice. A minority of patients may be diagnosed later in life, such as during adolescence. This progressive disease ultimately leads to severe cirrhosis and liver failure. Patients typically have to get a liver transplant in order to survive. In some variants, liver cancer may appear when patients are only one or two years old. To learn more about PFIC, click here.

There are currently no approved treatments specifically for PFIC but it is estimated to affect up to 100,000 children worldwide. The data from previous trials of A4250 have been encouraging so far. In Phase 2 trials, A4250 was able to significantly reduce itching and the concentration of serum bile acids. The Phase 3 trial will involve a total of 60 patients between the ages of six months and 18 years. All patients have either the subtype 1 or 2 variant of PFIC.

A4250 has also received orphan drug designations from both the FDA and the EMA, and also is participating in the EMA’s PRIority MEdicines (PRIME) program. These designations will accelerate the approval process for the drug if trials continue to go well and will also give Albireo a period of market exclusivity if the drug gets approved, guaranteeing a return on investment. A4250 is classified as an ileal bile acid transporter inhibitor. It acts locally in the digestive tract and therefore has minimal exposure to the rest of the body, which reduces potential safety concerns.

Hopefully, this new treatment will reach approval and provide a more effective treatment option for children with PFIC.

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