Zektayos - Hepjuvo: Withdrawal of the marketing authorisation application

Zektayos-Hepjuvo was developed as a medicine to treat adults with non-alcoholic steatohepatitis, an inflammation of the liver caused by a build-up of fat, that has already led to the development of some scarring of the liver (fibrosis) but not yet to cirrhosis (severe liver scarring).

Zektayos-Hepjuvo contains the active substance obeticholic acid and was to be available as tablets.

The active substance in Zektayos-Hepjuvo, obeticholic acid, is a modified form of a bile acid (the main components of bile, the digestive fluid that is produced by the liver). It works mainly by attaching to receptors (targets) in the gut and liver called farnesoid X receptors. Attachment to these receptors reduces the production of signals that lead to inflammation and scarring.

The company presented results from 931 patients with non-alcoholic steatohepatitis that had led to the development of fibrosis of the liver but not yet to cirrhosis, included in a main study that is ongoing. The main measures of effectiveness were the reduction of scarring and inflammation compared with placebo (a dummy treatment).

Based on the review of the data, at the time of the withdrawal, the Agency had recommended refusing marketing authorisation for Zektayos-Hepjuvo for the treatment of non-alcoholic steatohepatitis with fibrosis.

The Agency considered that the results of the study were not sufficient to establish the effectiveness of the medicine, with results showing only a small improvement when compared with placebo. In addition, some side effects of the medicine were considered of relevant concern as they increase risk for cardiovascular diseases (problems with the heart and blood vessels) and seem to also affect kidney function. Therefore, at the time of the withdrawal, the Agency’s opinion was that the benefits of Zektayos-Hepjuvo did not outweigh its risks.

In its letter notifying the Agency of the withdrawal of the application, the company stated that they were not able to satisfactorily address the Agency’s concerns in an acceptable timeframe.

The company informed the Agency that there are no consequences for patients in clinical trials using Zektayos-Hepjuvo.

If you are in a clinical trial and need more information about your treatment, speak with your clinical trial doctor.