EU/3/16/1748 - orphan designation for treatment of acute myeloid leukaemia

crenolanib besilate

Overview

On 14 October 2016, orphan designation (EU/3/16/1748) was granted by the European Commission to Arog Pharmaceuticals Europe Ltd, Ireland, for crenolanib besylate for the treatment of acute myeloid leukaemia.

What is acute myeloid leukaemia?

Acute myeloid leukaemia (AML) is a cancer of the white blood cells (cells that fight against infections). In patients with AML, the bone marrow (the spongy tissue inside the large bones, where blood cells are produced) produces large numbers of abnormal, immature white blood cells. These abnormal cells quickly build up in large numbers in the bone marrow and are found in the blood.

AML is a long-term debilitating and life-threatening disease because these abnormal immature cells take the place of the normal blood cells, causing bleeding episodes, blood clots and a reduced ability to fight infections.

What is the estimated number of patients affected by the condition?

At the time of designation, AML affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 51,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

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* Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 513,700,000 (Eurostat 2016).

What treatments are available?

Treatment for AML is complex and depends on a number of factors including the extent of the disease, whether it has been treated before, and the patient's age, symptoms and general state of health. At the time of designation, the main treatments for AML were chemotherapy (medicines to treat cancer) and haematopoietic (blood) stem-cell transplantation (where the patient's bone marrow is replaced by stem cells to form new bone marrow that can produce healthy blood cells).

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with AML, because early data showed that there was no evidence of disease after treatment with the medicine in patients whose disease did not respond to other treatments or had come back. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Crenolanib besylate is a 'tyrosine-kinase inhibitor'. This means that it blocks the activity of enzymes known as tyrosine kinases found in certain receptors in cancer cells. This medicine blocks the FLT3 receptor, which is involved in stimulating the AML cells to divide uncontrollably. By blocking this receptor, crenolanib besylate is expected to stop cell division and lead to cell death, and thus slow down the progression of the disease.

What is the stage of development of this medicine?

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with AML were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for AML. Orphan designation of the medicine had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 September 2016 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/16/1748: Public summary of positive opinion for orphan designation of crenolanib besylate for the treatment of acute myeloid leukaemia

Adopted Reference Number: EMA/629727/2016

Key facts

• Active substance:
  crenolanib besilate
• Intended use:
  Treatment of acute myeloid leukaemia
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/16/1748
• Date of designation:
  14/10/2016
• Sponsor:

  Arog Pharmaceuticals Europe Ltd; C/O TMF Group; 3rd floor, Kilmore House; Park Lane, Spencer Dock; Dublin 1; Ireland; Tel. [phone]; Fax [phone]; E-mail: [e-mail]