The U.S. Food and Drug Administration has granted the experimental drug A4250 rare paediatric disease designation for the treatment of progressive familial intrahepatic cholestasis. The full article can be found here, at BioPortfolio.
Progressive familial intrahepatic cholestasis (PFIC) is a condition characterised by progressive liver disease. It is caused by liver cells that do not secrete a normal amount of bile, causing a build-up of the substance inside the cells that, over time, leads to liver damage. PFIC is estimated to affect one in every 50,000 to 100,000 people worldwide. People who are affected by the condition may experience itchiness and jaundice, and symptoms typically first appear in childhood. There are three types of PFIC, which are each distinguished by different genetic causes, and tend to be associated with different effects.
An experimental drug called A4250 is being developed by Albireo Pharma to treat PFIC. A4250 acts as an ileal bile acid transporter inhibitor that has effects largely concentrated in the gut. It is being investigated in a Phase 3 clinical trial at the moment that will evaluate the effects and safety of the drug on sixty patients between 6 months and 18 years old with PFIC types 1 or 2.
The decision of the FDA to grant the drug rare paediatric disease designation is likely to lead to benefits for the developing company, Albireo, and is hoped to support the process of bringing the drug to market. The designation is reserved for drugs that address serious diseases that affect under 200,000 people aged between 0 and 18 in the US.