The first Nash drug in the world! Ocaliva (OCA, obacholic acid) enters the priority review in the United States, or will be approved for listing in the first quarter of 2020!

December 10, 2019 news / BIOON / -- intercept Pharma is a biopharmaceutical company focusing on the development and commercialization of new therapies for the treatment of progressive, non viral liver diseases Recently, the company announced that the positive results of the mid-term analysis of the phase III regenerate study to evaluate the treatment of fibrosis caused by nonalcoholic steatohepatitis (NASH) by ocaliva (obeticolic acid, OCA) have been published in the lancet It is worth mentioning that this is the first peer review publication to evaluate the positive results of key clinical studies of Nash treated by a research drug At the end of November this year, the new drug application (NDA) for the treatment of Nash induced fibrosis by OCA was accepted by the U.S FDA and granted priority review FDA gives priority to drugs that have the potential to treat serious diseases, and if approved, will provide significant progress in safety or effectiveness FDA has designated the target date of prescription drug user fee Act (PDUFA) for this NDA as March 26, 2020 In the United States, Nash is expected to be the main cause of liver transplantation as soon as 2020 If approved, OCA will be the first treatment available for patients with liver fibrosis caused by Nash It is worth mentioning that in NASH, OCA is the only research drug granted breakthrough drug qualification (BTD) by FDA, and it is the first one in the world to enter and successfully complete phase III clinical research drug The regenerate study was conducted in patients with stage 2 or stage 3 liver fibrosis due to Nash and evaluated the efficacy and safety of two doses of OCA (10mg and 25mg once a day) relative to placebo In the primary efficacy analysis, at the pre planned mid-term analysis of 18 months, the primary end point of fibrosis improvement (≥ 1 stage) and no deterioration of Nash was achieved at a dose of 25mg 0ca once a day compared with placebo (P = 0.0002) In addition, a higher proportion of patients in the 25 mg OCA group achieved the primary end point of Nash elimination and no deterioration of liver fibrosis compared with the placebo group The study is currently in progress and will continue to pass clinical results to verify and describe clinical benefits The 18 month interim analysis data of the study served as the basis for the application of new drugs (NDA) for the treatment of Nash induced fibrosis by OCA As mentioned earlier, intercept also plans to submit a marketing authorization application (MAA) to the European Drug Administration (EMA) in the fourth quarter of 2019 Zobair M younossi, professor and head of the Department of medicine, chairman of the regenerate Steering Committee and the first author of the article, said: "the first positive phase III study in NASH represents a real watershed in this field of Hepatology The antifibrotic effect observed in the regenerate study after 18 months of OCA treatment is particularly significant, as fibrosis is the most important histological predictor of liver failure and death in patients with NASH " Octalva is a farneside X receptor (FXR) agonist, which is a nuclear receptor expressed in liver and small intestine, and a key regulator in bile acid, inflammation, fibrosis and metabolic pathways In the United States, ocaliva was approved for marketing in May 2016 for the treatment of primary biliary cholangitis (PBC) At present, ocaliva is being developed for the treatment of many other chronic liver diseases, including Nash, primary sclerosing cholangitis, biliary atresia, etc Nonalcoholic steatohepatitis (NASH) is a kind of serious progressive liver disease Chronic inflammation caused by excessive accumulation of liver fat leads to progressive fibrosis (scar), which can lead to cirrhosis, liver failure, liver cancer and death Late fibrosis is associated with a marked increase in liver incidence rate and mortality in NASH patients According to nature, Nash has become the second most common cause of liver transplantation after chronic hepatitis C in the United States and is expected to be the leading cause in 2020 At present, the market size of Nash has reached US $40 billion However, despite the urgent medical needs, so far no drug has been approved for NASH treatment Original source: 1 Intercept announcements publication of results from the interim analysis of the phase 3 generate study of OCA for the treatment of fibrosis due to NASH in the language 2 Objective acid for the treatment of non-alcoholic steatohepatis: interim analysis from a multicenter, random, placebo controlled phase 3 trial