Vynfinit: Withdrawal of the marketing authorisation application

Vynfinit is a medicine that contains the active substance vintafolide. It was to be available as a powder to be made up into a solution for injection.

Vynfinit was to be used in combination with pegylated liposomal doxorubicin (PLD, another cancer medicine) to treat adult patients with ovarian cancer which has become resistant to platinum-based cancer treatment.

Vynfinit was intended for use in women whose ovarian cancer cells have high levels of proteins called folate receptors present on their surface. Before starting Vynfinit, the presence of these folate receptors was to be detected in all tumours using an approved diagnostic medicine.

Vynfinit was designated an 'orphan medicine' (a medicine to be used in rare diseases) on 9 February 2012 for the treatment of ovarian cancer.

The active substance in Vynfinit, vintafolide, is made up of two components, desacetylvinblastine hydrazide (a medicine that kills cells that are dividing, such as cancer cells) and folic acid (a molecule that targets folate receptors, which are found at high levels on certain cancer cells). The folic acid component of the medicine delivers the medicine specifically to cells with folate receptors, attaching to them and allowing the medicine to enter the cell. Once inside the cell, desacetylvinblastine hydrazide is released and attaches to a protein called tubulin, which is important for the formation of the internal 'skeleton' that cells need to assemble when they divide. By attaching to tubulin in cancer cells, desacetylvinblastine hydrazide stops the formation of the skeleton, preventing the division and spread of the cancer cells.

Vynfinit was investigated in one main study involving 162 patients whose cancer had become resistant to platinum-based treatment and who received PLD with or without Vynfinit. The main measure of effectiveness was how long patients lived without their disease getting worse.

The evaluation had finished and the CHMP had recommended a conditional marketing authorisation for Vynfinit. After authorisation the company was to provide confirmatory data from an ongoing study with Vynfinit. However, before the authorisation process could be completed by the European Commission, preliminary data from this study became available which showed that the study could not confirm the benefit of Vynfinit in ovarian cancer patients. Therefore the company had to terminate the study and decided to withdraw the application.

Following the termination of the study, the European Commission had requested the CHMP to revise its recommendation but the application was withdrawn before the CHMP had started the revision. However, although no formal review has been carried out, the CHMP informed the European Commission that, as confirmatory data on the benefits of Vynfinit will not be forthcoming, the grounds for the previous CHMP recommendation for a conditional marketing authorisation are no longer valid.

In its letter notifying the Agency of the withdrawal of application, the company stated that the condition of the marketing authorisation for Vynfinit to provide confirmatory data will not be met since the study had been terminated. The preliminary data from the study could not confirm the benefit of Vynfinit in ovarian cancer patients.

In addition the company also notified the Agency of the withdrawal of Folcepri and Neocepri, two medicines which were to be used before treatment with Vynfinit in order to determine whether patients would be suitable for treatment with Vynfinit.

The company informed the CHMP that patients who were enrolled in the study that was terminated will be able to decide whether to continue treatment. Patients who are currently in a compassionate use programme may be able to receive further treatment. Patients who need any information should speak to their doctor.