EMA Recommends Refusal of a Change to the Marketing Authorisation for Eltrombopag

The change concerned an extension of indications to previously untreated patients with severe aplastic anaemia from 2 years of age
06 Aug 2019
Haematologic malignancies;  Cancer in Special Situations / Population

The European Medicines Agency (EMA) has recommended the refusal of a change to the marketing authorisation for eltrombopag (Revolade). The change concerned an extension of indication to add treatment of previously untreated patients with severe aplastic anaemia from 2 years of age.

The EMA issued this opinion on 27 June 2019. The company that applied for the change to the authorisation, Novartis Europharm Limited has requested a re-examination of the opinion. Subsequently, EMA has issued an update on 25 July 2019 by stating that upon receipt of the grounds of the request, it will re-examine its opinion and issue a final recommendation.

Revolade is a medicine used to treat:

  • patients from 1 year of age with immune thrombocytopenia, if other usual treatment has not worked;
  • thrombocytopenia in adults with chronic (long-term) hepatitis C when the disease prevents the use of certain types of hepatitis C treatment;
  • adults with severe aplastic anaemia, if immunosuppressive therapy has not worked and the patient cannot receive bone marrow transplantation.

Revolade has been authorised in the EU since March 2010. It contains the active substance eltrombopag and is available as tablets and as a powder to make up a liquid to take by mouth.

Further information on Revolade’s uses can be found on the Agency’s website.

The company applied for an extension of indication to add treatment of severe aplastic anaemia in previously untreated adults and children aged from 2 years.

In the body, a hormone called thrombopoietin stimulates the production of platelets and some types of blood cells by attaching to certain receptors in the bone marrow. The active substance in Revolade, eltrombopag, also attaches to and stimulates the thrombopoietin receptors. This increases the production of platelets and some other blood cells, improving platelet and blood cell counts.

To support its application, the company provided data from a study involving 153 patients from 3 years of age with severe aplastic anaemia who had not previously received immunosuppressive therapy. In the study, Revolade was combined with immunosuppressants that are used for treating aplastic anaemia. Treatment was considered successful if the patient’s white blood cell and platelet counts and haemoglobin rose to satisfactory levels.

The design of the study was not considered sufficiently robust to show that Revolade is effective for treating severe aplastic anaemia in previously untreated patients. The study did not make a direct comparison between Revolade combined with immunosuppressant treatment and immunosuppressant treatment alone. Instead, the comparison was with patients treated with immunosuppressants in other studies. Such comparison prevents drawing reliable conclusions on the effect of Revolade when added to immunosuppressants. Moreover, adequate amount of data were not available on the use of Revolade in children.

Therefore, the Agency’s opinion was that the balance of benefits and risks of Revolade in the initial treatment of severe aplastic anaemia could not be established. Hence, the Agency recommended refusing the change to the marketing authorisation.

The company informed the Agency that there are no consequences for patients who are receiving Revolade for initial treatment of severe aplastic anaemia in clinical trials or in compassionate use programmes.

There are no consequences for Revolade in its authorised uses.

Last update: 06 Aug 2019

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