EU/3/16/1758

On 14 October 2016, orphan designation (EU/3/16/1758) was granted by the European Commission to Clinical Network Services (UK) Ltd, United Kingdom, for P-ethoxy growth factor receptor-bound protein 2 antisense oligonucleotide 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 1.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 77,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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. Early studies showed a favourable response when the medicine was used in combination with a currently authorised product in patients whose disease had come back despite previous treatment. 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?

This medicine is an ‘antisense oligonucleotide’, a small strand of synthetic genetic material. It has been designed to attach to the gene for the protein growth factor receptor-bound protein 2 (Grb2), thereby blocking its production. Grb2 is involved in the uncontrolled growth of cancer cells including AML cells. By blocking the production of Grb2, the medicine is expected to stop the cancer cells growing, 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.