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A4250 for progressive familial intrahepatic cholestasis


Gastrointestinal, Pancreatic and Liver Disease

September 2017

Progressive familial intrahepatic cholestasis (PFIC) is a rare, inherited condition that usually begins in infancy. The condition affects the liver, hindering or stopping the flow of bile from the liver. Bile flow is needed for fats, nutrients and vitamins to be absorbed into the body, and also to help the body get rid of toxins. Problems absorbing fats and nutrients can lead to poor weight gain and slower growth, and excess toxins in the body can lead to jaundice and itching, which can have a large impact on the quality of life of the patient and their family. There is no cure for this condition, and current treatments can only reduce the symptoms and slow down damage to the liver. It is not known how many people have this condition, but around 35 genetic tests are carried out each year for this condition.
The drug A4250 works in a new way that directly targets the part of the gut that allows bile to flow into the liver. By stopping bile from coming into the liver, bile levels are reduced, slowing down damage to the liver and reducing the symptoms of jaundice and itching. Because the drug acts directly on the gut, it has the potential to reduce the side-effects that occur with current medicines, such as problems with vitamin absorption. The drug is taken by mouth as a capsule.

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