Experimental Treatment for Biliary Atresia Gets Orphan Drug Designation in the EU

According to a story from Market Screener, the drug developer Albireo Pharma, Inc., recently announced that its investigational product candidate A4250 has earned Orphan Drug designation from the European Commission. This designation was granted for the treatment of the rare lung disease biliary atresia. Albireo is dedicated to the development of bile acid modulators to treat rare, pediatric diseases of the liver.

About Biliary Atresia

Biliary atresia is an illness in which at least one of the bile ducts in the liver has become blocked, abnormally narrow, or is absent altogether. There are multiple possible causes of biliary atresia. In many cases, the cause is not known, but it is sometimes associated with other abnormalities; genetic changes affecting the ADD3 gene has been identified as the cause in some cases. Other causes include exposure to aflatoxin B1 or B2 during pregnancy. There are three different types of biliary atresia which are distinguished by which ducts are affected. Symptoms include jaundice, cholestatsis, itching, pale stool, poor nutrient absorption, abdominal swelling, dark urine, liver failure, and cirrhosis. Treatment includes surgery, corticosteroids, and liver transplant. Females and people of Asian or African-American descent are more likely to be affected. To learn more about biliary atresia, click here.

Orphan Drug Designation in The EU

Orphan Drug designation in the EU is intended of rare and life threatening diseases. A rare disease is considered one that affects fewer than 5 in every 10,000 people in the EU. To qualify, a drug must display potential advantages in effectiveness or safety over currently available treatments. Alternatively, it must fulfill a medical need that is not satisfied by current treatments. Orphan designation confers several benefits to the recipient company, such as more streamlined regulatory processes and registration, protocol assistance, and a ten year period of market exclusivity if the therapy gains approval. Completion of a pediatric investigation plan could allow two additional years of exclusivity for pediatric therapies.

About A4250

A4250 is classified as a ileal bile acid transporter inhibitor. It already hold multiple orphan designations in both the US and EU for treating other rare diseases of the liver, such as primary biliary cholangitis, progressive familial intrahepatic cholestasis, and Alagille syndrome.


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