On July 20, 2021, the US FDA approved the listing of Bylvay developed by Albireo Pharma through fast track and orphan drugs. This is the world's first drug used to treat itchy skin caused by progressive familial intrahepatic cholestasis.

Progressive familial intrahepatic cholestasis (PFIC) is a group of autosomal recessive inherited diseases, with intrahepatic cholestasis as the main manifestation, usually onset in infancy or childhood. According to different pathogenic genes, PFIC can be divided into 6 types. If these children do not receive timely intervention, they usually develop liver cirrhosis and liver failure in childhood. For the treatment of PFIC, only surgery (biliary shunt or liver transplantation) can be used. It is the only effective method for end-stage PFIC liver transplantation. Treatment methods, otherwise it will be difficult for most patients to live beyond 30 years of age.

The approval of Bylvay is undoubtedly the gospel for the majority of PFIC. This is the world's first drug approved for the treatment of all subtypes of PFIC. It also represents a complete change in the treatment of PFIC and successfully subverted the clinical practice of PFIC children. The treatment model is an important milestone in the field of PFIC treatment.

Bylvay is developed by Albireo Pharma. The active ingredient is odevixibat, which is a first-ever potent, selective, non-systemic, ileal bile acid transporter (IBAT) inhibitor with minimal systemic exposure and a local effect in the intestine. It can be used for the treatment of rare childhood cholestatic liver diseases, including PFIC, biliary atresia, and Alagille syndrome (ALGS), of which PFIC is the first target indication. Bylvay does not require refrigeration and can be taken in capsule form once a day.

The FDA's approval of Bylvay is based on the results of two major clinical trials. The results of clinical data show that Bylvay can attenuate the symptoms of skin itching in children and reduce the concentration of bile acids in the serum. Bylvay is well tolerated clinically, and the most common adverse reactions are diarrhea and frequent bowel movements. At present, odevixibat, as the first in class for the treatment of PFIC, is approved for marketing in the European Union and the United States, and will soon be listed in Australia, Canada, Israel and other regions.