Vanflyta

Vanflyta can only be obtained with a prescription and treatment should be started by a doctor experienced in using cancer treatments.

Before taking Vanflyta, the patient must have a test to confirm that their cancer cells have the ITD mutation in the FLT3 gene (ITD-FLT3 positive).

Vanflyta is available as tablets to be taken by mouth. It is taken once a day for two weeks during each 4-week chemotherapy cycle. After chemotherapy is complete, Vanflyta is taken once a day on its own as maintenance treatment. Treatment can continue for up to 36 cycles lasting 4 weeks each.

For more information about using Vanflyta, see the package leaflet or contact your doctor or pharmacist.

A main study involving 539 patients newly diagnosed with AML compared Vanflyta with placebo (a dummy treatment). Patients were given Vanflyta or placebo in combination with chemotherapy. Those whose cancer responded to treatment either continued their treatment without chemotherapy or had a blood stem cell transplant before continuing their treatment. After three years of treatment, 50% of patients who received Vanflyta were still alive compared with 41% of those who received placebo.

For the full list of side effects and restrictions with Vanflyta, see the package leaflet.

The most common side effects with Vanflyta (which may affect more than 1 in 5 people) include increased levels of an enzyme in the blood called alanine aminotransferase, decreased levels of blood platelets, decreased levels of haemoglobin (the protein in red blood cells that carries oxygen around the body), diarrhoea, nausea (feeling sick), abdominal (belly) pain, headache, vomiting and decreased levels of neutrophils (a type of white blood cell).

The most common serious side effect with Vanflyta (which may affect up more than 1 in 10 people) is neutropenia (low levels of neutrophils). Other common serious side effects (which may affect up to 1 in 10 people) include fungal infections and herpes infections.

The most common serious side effects with Vanflyta (which may affect up to 1 in 10 people) that led to dose reduction or interruption include neutropenia, thrombocytopenia (low levels of blood platelets) and prolonged QT interval (abnormal electrical activity of the heart that affects its rhythm).

Vanflyta should not be used by patients who are breast-feeding or who have congenital long QT syndrome (abnormal electrical activity of the heart caused by a gene defect).

When used in combination with chemotherapy, Vanflyta has been shown to prolong the lives of people newly diagnosed with ITD-FLT3-positive AML. Although some of the medicine’s side effects can be serious, the Agency considered that adequate measures were in place to manage or minimise the risks with Vanflyta.

The European Medicines Agency therefore decided that Vanflyta’s benefits are greater than its risks and it can be authorised for use in the EU.

The company that markets Vanflyta will provide educational materials for healthcare professionals and patients on how to minimise the risk of prolonged QT interval and recognise signs and symptoms of this side effect.

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Vanflyta have also been included in the summary of product characteristics and the package leaflet.

As for all medicines, data on the use of Vanflyta are continuously monitored. Suspected side effects reported with Vanflyta are carefully evaluated and any necessary action taken to protect patients.

Vanflyta received a marketing authorisation valid throughout the EU on 6 November 2023.

The active substance in Vanflyta, quizartinib, blocks the action of enzymes known as tyrosine kinases, in particular a tyrosine kinase called FLT3 that normally controls growth and division of white blood cells. In patients who have an FLT3 mutation, the FLT3 enzyme is overactive and stimulates the growth of too many white blood cells. By blocking FLT3, quizartinib is expected to stop the growth of white blood cells and thus slow down the development of the cancer.

Vanflyta was developed as a cancer medicine for the treatment of a type of AML called ‘FLT3-ITD positive’ (when the cancer cells have a certain change in the gene for a protein called FLT3). Vanflyta was to be used in adult patients whose disease had come back or did not respond to previous treatments and for continuation of treatment after the patient had undergone a haematopoietic stem cell transplant (HSCT, a transplant of cells that can develop into different types of blood cells).

Vanflyta contains the active substance quizartinib and was to be available as tablets.

Vanflyta was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 23 March 2009 for the treatment of AML. Further information on the orphan designation can be found on the Agency’s website.

The company presented the results of a study in 367 patients with FLT3-ITD positive AML whose disease did not respond to treatment or came back after treatment. Vanflyta was compared with other cancer medicines and the main measure of effectiveness was overall survival (how long patients lived) after being given Vanflyta or the comparator medicines.

Although the results from the main study indicated a marginal improvement in overall survival for patients given Vanflyta, the study had important limitations which meant that the effectiveness of Vanflyta could not be sufficiently demonstrated.

Therefore, the Agency’s opinion was that the benefits of Vanflyta did not outweigh its risks and it recommended refusing marketing authorisation.

The company informed the Agency that there are no consequences for patients in clinical trials or in compassionate use programmes with Vanflyta.

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