Restaysis: Withdrawal of the marketing authorisation application

Restaysis is a medicine that contains the active substance ciclosporin. It was to be available as eye drops.

Restaysis was expected to be used in adults with moderate dry eye disease that was inadequately controlled despite treatment with tear substitutes or eye lubricants.

Inflammation seems to have an important role in the development of dry eye disease, regardless of the cause of the disease.

The active substance in Restaysis, ciclosporin, reduces the activity of cells of the immune system (the body's natural defences) that are involved in the processes that cause inflammation. Applying it directly to the eye was expected to reduce inflammation and the symptoms of the disease.

The company presented an analysis of the results from three studies with ciclosporin conducted in the 1990s in patients with moderate to severe dry eye disease, where Restaysis was compared with placebo (a dummy treatment). The analysis focused on a subgroup of patients with moderate dry eye disease. The main measures of effectiveness were the number of patients with damaged eye surface and with blurred vision, after 6 months of treatment.

The application was withdrawn after the CHMP had evaluated the documentation provided by the company and formulated lists of questions. After the CHMP had assessed the company's responses to the last round of questions, there were still some unresolved issues.

Based on the review of the data and the company's response to the CHMP lists of questions, at the time of the withdrawal the CHMP had some concerns and was of the provisional opinion that Restaysis could not have been approved for the treatment of moderate dry eye disease.

The CHMP concluded that the results presented by the company were not robust enough to demonstrate that Restaysis is effective. In particular, the way the results were analysed in patients with moderate dry eye disease could have introduced a bias in favour of Restaysis.

Therefore, at the time of the withdrawal, the CHMP was of the opinion that, because of lack of proven effectiveness, the benefits of Restaysis did not outweigh its risks.

In its letter notifying the Agency of the withdrawal of the application, the company stated that it withdrew its application because of the CHMP's view that the data provided did not allow concluding on a positive benefit-risk balance in the intended patient population.

The withdrawal letter is available.

The company informed the CHMP that the withdrawal will have no consequences on ongoing clinical trials.

If you are in a clinical trial and need more information about your treatment, contact the doctor who is giving it to you.