Tasigna: Withdrawal of the application to change the marketing authorisation

Tasigna is a cancer medicine that contains the active substance nilotinib. It is available as capsules (150 and 200 mg).

Tasigna has been authorised in the EU since 19 November 2007. It is used to treat CML, a type of cancer of the white blood cells, in patients who have the Philadelphia chromosome (Ph+ CML). This means that patients produce an abnormal enzyme called Bcr-Abl kinase that leads to the development of the cancer.

Tasigna is used to treat the 'chronic' and 'accelerated' phases of CML in patients who have not responded to other treatments including imatinib or when they cannot tolerate these treatments. It is also used to treat newly diagnosed patients with CML in the chronic phase.

Tasigna was designated an 'orphan medicine' (a medicine to be used in rare diseases) on 22 May 2006 for CML.

Tasigna was also expected to be used to treat adults with Ph+ CML in the chronic phase in whom treatment with imatinib did not lead to a 'complete molecular response'. A complete molecular response is achieved when Bcr-Abl can no longer be detected in the patient's blood. This would have meant that patients who had only partially responded to treatment with imatinib could have been switched to Tasigna.

In the new indication, Tasigna was expected to work in the same way as it does in its existing indications. The active substance in Tasigna, nilotinib, is a 'tyrosine kinase inhibitor' which acts by blocking Bcr-Abl kinase, an abnormal enzyme which is produced by leukaemia cells and causes them to multiply uncontrollably. By blocking Bcr-Abl kinase, Tasigna helps to control the multiplication of leukaemia cells.

The company presented the results from one main study with Tasigna involving 207 patients with Ph+ CML in the chronic phase and who did not have a complete molecular response following imatinib treatment for at least two years. Patients in the study either received Tasigna or had further treatment with imatinib. The main measure of effectiveness was based on the percentage of patients who had a complete molecular response during the first 12 months of the study.

The application was withdrawn after the CHMP had evaluated the documentation provided by the company and formulated lists of questions.

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 Tasigna could not have been approved for the treatment of patients with Ph+ CML who did not have a complete molecular response following imatinib treatment. This was because the study failed to demonstrate that treatment with Tasigna significantly increased the percentage of patients who had a complete molecular response by 12 months. In addition, it was unclear how a complete molecular response would translate into an improvement of patient's outcome. The CHMP was also concerned that the dose of imatinib that was used in the study may have been too low. Regarding safety, more side effects were reported with Tasigna than with imatinib.

Therefore, at the time of the withdrawal, the CHMP was of the opinion that the benefits of Tasigna in this new indication did not outweigh its risks.

In its letter notifying the Agency of the withdrawal of application, the company stated that the withdrawal was due to the fact that data gathered so far are insufficient for the CHMP to recommend approval of the new indication.

The company informed the CHMP that there are no consequences for patients currently included in clinical trials using Tasigna.

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

There are no consequences on the use of Tasigna in its authorised indications.