Ontilyv - withdrawal of application for variation to marketing authorisation
Application withdrawn
The application for a change to this medicine's authorisation has been withdrawn
opicapone
Post-authorisationHuman
Ontilyv is a medicine used for treating adults with Parkinson’s disease, a progressive brain disorder that causes shaking and muscle stiffness, and slows movement.
Ontilyv is used as an add-on to levodopa / DOPA decarboxylase inhibitors (DDCI) (other medicines for Parkinson’s disease) in patients who are having motor fluctuations in the control of their condition. Fluctuations happen when the effects of the medication wear off and symptoms re-emerge before the next dose is due. Ontilyv is used when these fluctuations cannot be treated with the standard levodopa-containing combinations alone.
Ontilyv has been authorised in the EU since June 2016.
It contains the active substance opicapone and is available as capsules to be taken by mouth.
Further information on Ontilyv’s current uses can be found on the Agency’s website: ema.europa.eu/en/medicines/human/EPAR/Ontilyv
The company applied to extend the use of Ontilyv to treat signs and symptoms of Parkinson’s disease; this means that its use in addition to levodopa / DDCI was to be extended to a broader group of patients, including patients with early stage Parkinson’s disease. It was no longer to be restricted to patients having (motor) fluctuations in the control of their condition.
In patients with Parkinson’s disease, the cells in the brain that produce the neurotransmitter dopamine begin to die and the amount of dopamine in the brain decreases. The patients then lose their ability to control their movements reliably. The active substance in Ontilyv, opicapone, works to restore the levels of dopamine in the parts of the brain that control movement and coordination. It enhances the effects of levodopa, a copy of the neurotransmitter dopamine that can be taken by mouth. Opicapone blocks an enzyme that is involved in the breakdown of levodopa in the body called catechol-O-methyl transferase (COMT). As a result, levodopa remains active for longer. This helps to improve the symptoms of Parkinson’s disease, such as stiffness and slowness of movement.
The company presented data from a study involving 355 patients with early stage Parkinson’s disease who received either Ontilyv or placebo (a dummy treatment) in addition to levodopa / DDCI. Patients in the study had no signs of any motor complications (meaning fluctuations in their response to treatment or involuntary movements). The main measure of effectiveness was an improvement in motor symptoms as measured using a standard scale called Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS).
The application was withdrawn after the European Medicines Agency had evaluated the information from the company and had prepared questions for the company. After the Agency had assessed the company’s responses to the questions, there were still some unresolved issues.
Based on the review of the information and the company’s response to the Agency’s questions, at the time of the withdrawal, the Agency had some concerns and its provisional opinion was that Ontilyv could not have been authorised for the treatment of signs and symptoms of early stage Parkinson’s disease.
The Agency noted that the data on the effectiveness of Ontilyv were not robust enough and indicated only a small effect of the medicine on MDS-UPDRS score and other measures of effectiveness. It was therefore not clear whether patients with mild symptoms of early stage Parkinson’s disease would benefit from treatment with Ontilyv.
Therefore, at the time of the withdrawal, the Agency’s opinion was that, because effectiveness was not proven in the broader use, the benefits of Ontilyv did not outweigh its risks.
In its letter notifying the Agency of the withdrawal of application, the company stated that they withdrew their application due to the need to conduct additional work to fully address the questions raised by the Agency.
The company informed the Agency that there are no consequences for patients in clinical trials using Ontilyv.
If you are in a clinical trial and need more information about your treatment, speak with your clinical trial doctor.
There are no consequences on the use of Ontilyv in its authorised uses.