A study recently published in the prestigious journal The New England Journal of Medicine, of which has been co-author Dr. Victor Vargas, head of the Section of the Haepatology Service of the Vall d'Hebron University Hospital and researcher at the Liver Diseases Research Group at Vall d'Hebron Research Institute (VHIR), has proven the effectiveness of the acid obeticholic to treat primary biliary cholangitis (PBC), a liver disease hitherto known as primary biliary cirrhosis.
The study, a phase 3 clinical study, double-blind, placebo-controlled, analyzed the results of this treatment in 217 patients from 59 centers in 13 countries for 12 months. The results are very promising, considering that until now there was only one drug to stop the development of this disease, ursodeoxycholic acid, which has no effect on a large number of patients (between 40 and 50%). These results have been a key factor for the acid obeticholic to receive accelerated approval from the Food and Drug Agency of the United States (FDA) as a treatment of PBC in adult patients who have no response or do not tolerate ursodeoxycholic acid.
The researchers divided the patients into three groups. They all had PBC and at the same time a poor response to the treatment or presented unacceptable side effects to ursodeoxycholic acid. The first group received a dose of 10 mg of obeticholic acid, the second 5 mg, 10 mg adjustable if necessary, or placebo. The aim was to improve the biochemical markers of the disease, bilirubin and alkaline phosphatase. In particular, maintaining normal levels of bilirubin and alkaline phosphatase less than 1.67 times above the upper limit of the normal level.
The article notes that, according to the study, the addition of obeticholic acid to treatment with ursodeoxycholic acid get an improvement of these markers. In patients in the first group, 47% improved, and in the second, 46% did. Among those who received placebo only 10% did. However, most side effects were detected in those who had received treatment, but in any case they left sequelae. Now will begin a phase 3b clinical study to analyze its effect on mortality and clinical outcomes.
Primary biliary cholangitis
Primary biliary cholangitis, formerly known as primary biliary cirrhosis, is an autoimmune disease, one of the so-called rare diseases, that affects the liver. Its progress is slow. It causes destruction of the intralobulars bile ducts by lymphocytes. Its evolution may end in terminal cirrhosis or liver failure, forcing the patient to undergo, if there is no treatment, a liver transplant.
It has a prevalence between 20 and 40 cases for every 100,000 people and affects mostly women. The high levels of alkaline phosphatase and bilirubin increase are related to the progression of the disease. So far only one treatment was known, the ursodeoxycholic acid.
The obeticholic acid
The obeticholic acid is a derivative from the chenodeoxycholic acid, a bile acid. It is a nuclear receptor agonist and has become a drug with good prospects in the treatment of various liver diseases.
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