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Two drugs approved by the EMA

written by

Leukaemia Care, Charity

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The European Medicines Agency (EMA) is responsible for the scientific evaluation of medicines developed by pharmaceutical companies for use in the European Union (EU), in order to safeguard public health and to enable access of European patients to innovative drugs.

The EMA has recently approved the use of eltrombopag, a once-daily oral tablet marketed by Novartis as Revolade®, as a treatment option for patients with the blood disorder severe aplastic anaemia (SAA).

SAA is a rare disorder in which the bone marrow fails to make enough blood cells. It leaves patients at risk from serious, life-threatening infections or bleeding. Two out of every one million people in Europe are diagnosed with the condition every year, with around 125 new cases in the UK each year.

The EMA recommends the treatment indication for patients who failed to respond to immunosuppressive therapy (IST), are heavily pre-treated or are unsuitable for stem cell transplantation. It is the first approved therapy in the EU for SAA patients who have not responded to alternative treatments. Approval came following the results of Phase II trial data showing a haematologic response (40%) in SAA patients when treated with eltrombopag who had not responded to IST. 


The EMA has also approved panobinostat as a therapy option for relapsed or refractory myeloma patients. Panobinostat is marketed by Novartis as Farydak®.

Multiple myeloma is a rare cancer which affects the plasma cells, a type of white blood cell found in the bone marrow (the spongy tissue found inside the bones). The most common symptom of myeloma is bone pain, especially in the spine but other bones may be affected such as the ribs, skull or pelvis. Each year there are approximately 39,000 people diagnosed with myeloma in the EU, with around 4,792 new cases in the UK annually.

Panobinostat has been recommended to be administered alongside bortezomib and dexamethasone for relapsed and/or refractory myeloma patients who have previously received at least two therapies.  Approval of the drug followed results of clinical data which showed that it slowed the progression of myeloma.

How does this affect NHS patients?

This approval allows for the marketing authorisation of eltrombopag and panobinostat throughout Europe for the recommended indications.  However, before both drugs can be prescribed to NHS patients in the UK, they will require appraisal and need approval from the relevant UK health technology appraisal (HTA) bodies.

The National Institute for Health and Care Excellence (NICE) HTA programme develops guidance on the use of new and existing medicines and therefore decides what treatments should be available to patients via the NHS. NICE guidance applies in England and sometimes also in Wales.

There are two additional UK bodies which conduct similar processes. In Scotland, assessments of the clinical and cost effectiveness of all new drugs are conducted by the Scottish Medicines Consortium (SMC) and in Wales, the All Wales Medicines Strategy Group (AWMSG) undertakes assessment of new drugs, particularly when NICE has decided not to review, has reviewed and not recommended or will not be reviewing the drug for a significant period of time.

If you could be affected by any of these changes and would like to speak to somebody, please call our CARE line on 08088 010 444.