Summary of Odevixibat indications and countries where it is marketed

Odevixibat (Odevixibat) is an oral small molecule drug that is a selective intestinal bile acid transporter inhibitor (IBAT inhibitor). It mainly inhibits the reabsorption of bile acids in the ileum, thereby reducing the concentration of bile acids in plasma and liver, thereby achieving the purpose of alleviating the symptoms of hepatobiliary diseases. The drug is mainly used to treat progressive familial intrahepatic cholestasis (PFIC), a rare hepatobiliary system disease in children, especially patients with PFIC1 and PFIC2 types. It can effectively relieve itching, reduce bile acid levels and improve quality of life.

Orvitixibate is produced by Swedish companyAlbireo Pharma developed it and obtained it for the first time in the United States in 2021 under the trade name of BylvayFDAapproved the marketing for the treatment of children 3 months old and above suffering from PFIC. Subsequently, the European Medicines Agency (EMA) also approved its marketing in EU member states for the treatment of PFIC children 6 months and older. Orvisibat thus became the world's first oral drug approved for the treatment of PFIC, marking a major breakthrough in the treatment of rare cholestatic diseases.

In addition to the United States and the European Union, orvisibate has also successively obtained marketing qualifications in many countries or regions including the United Kingdom, Canada, Israel, the United Arab Emirates, Australia, Brazil, and Singapore. Especially in European and American countries, because its treatment scope is concentrated in the field of rare childhood diseases, local regulatory agencies usually grant "orphan drug" status, rapid approval and market exclusivity period support, further accelerating its global launch process. Up to now, research on the indications of orvixibat is continuously expanding to include other types of cholestatic liver disease, such as Alagille syndrome and infantile cholestasis.

It should be pointed out that orvixibat has not yet been officially launched in mainland China. If domestic patients need to use the drug, they still need to obtain it through overseas medical institutions or cross-border drug service channels. At the same time, because the drug is positioned as a drug for rare diseases, its price is relatively high, and it is not yet included in the scope of medical insurance in China. In the future, as international multi-center clinical trials continue to advance, the drug is expected to enter the market in more countries and bring treatment hope to more children with cholestasis.

Reference materials:https://www.drugs.com/