PFIC is a group of diseases characterized by lack of proper flow of bile (the fluid that help with the digestion of fats in the small intestines). Because of mutations in key proteins in PFIC patients, bile is accumulating in the liver causing considerable damage to the organ. Many patients present with liver failure at young age, making liver transplant a necessity. In addition, like many other chronic liver conditions, PFIC patients often experience pruritis or itch than can have a severe impact on quality of life.

There are currently no medicines approved for the treatment of PFIC and besides liver transplant, an invasive surgical procedure to enable external drainage of bile is the only treatment option thus far in the management of PFIC.

In the clinical study results that were released by Albireo, odevixibat had had a profound effect on all major clinical endpoints defined in the study versus placebo. Most importantly, the group of PFIC patients that were given odevixibat saw significant decreases in bile levels and also significantly lower levels of pruritus.

Besides handing in new drug applications to the FDA in the US, EMA in Europe and other regulatory agencies across the world for the use of odevixibat as treatment for PFIC, Albireo is also conducting clinical trials across many so called cholestatic diseases in children where excess bile play a role. In the two days since the data announcement, the Abireo share price has increased by 52%.