EMA Recommends Refusal of the Marketing Authorisation for Quizartinib

It was developed for the treatment of patients with FLT3-ITD positive AML
22 Oct 2019
Anticancer agents & Biologic therapy;  Haematologic malignancies;  Personalised medicine

The European Medicines Agency (EMA) has recommended the refusal of the marketing authorisation for quizartinib (Vanflyta), a medicine intended for the treatment of adults with acute myeloid leukaemia (AML). The Agency issued its opinion on 17 October 2019.

The company that applied for authorisation, Daiichi Sankyo Europe GmbH, may ask for re-examination of the opinion within 15 days of receiving the opinion.

What is Vanflyta and what was it intended to be used for?

Vanflyta was developed as a cancer medicine for the treatment of FLT3-ITD positive AML. It was to be used in adult patients whose disease recurred or did not respond to previous treatments and for continuation of treatment after the patient had undergone a haematopoietic stem cell transplant (HSCT).

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.

How does Vanflyta work?

The active substance in Vanflyta, quizartinib, is a receptor tyrosine kinase inhibitor. It works by blocking FLT3, a protein involved in cell growth and proliferation. By blocking FLT3, quizartinib is expected to stop cancer cells from multiplying and thus slow down the progression of the disease.

What did the company present to support its application?

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

What were the main reasons for refusing the marketing authorisation?

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.

Does this refusal affect patients in clinical trials or compassionate use programmes?

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

Last update: 22 Oct 2019

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