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On 22 May 2017, orphan designation (EU/3/17/1876) was granted by the European Commission to TMC Pharma Services Ltd, United Kingdom, for sodium (1R,3R,4R,5S)-3-({2-N-acetylamino-2-deoxy-3-O-[(1S)-1-carboxylato-2-cyclohexylethyl]-beta-D-galactopyranosyl}oxy)-4-({6-deoxy-alpha-L-galactopyranosyl}oxy)-5-ethyl-cyclohexan-1-yl-(38-oxo-2,5,8,11,14,17,20,23,26,29,32,35-dodecaoxa-39-azahentetracontan-41-yl)carboxamide (also known as GMI-1271) for the treatment of acute myeloid leukaemia.
The sponsorship was transferred to FGK Representative Service GmbH, Germany in March 2018.
Acute myeloid leukaemia (AML) is a cancer of the white blood cells (cells that fight 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.
At the time of designation, AML affected approximately 1.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 67,000 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).
*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 515,700,000 (Eurostat 2017).
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 (a procedure where the patient's bone marrow is cleared of cells and replaced by stem cells to form new bone marrow that produces healthy blood cells).
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with AML because early studies have shown improvement in patients whose disease had not got better with other treatment or came back after treatment. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
In patients with AML, the AML cancer cells attach to a substance called E-selectin which is present in the bone marrow. This attachment allows AML cells to resist the effects of chemotherapy and encourages build-up of the cells in the bone marrow. The medicine blocks the action of E-selectin. This is expected to cause the AML cells to move out of the bone marrow and make them more susceptible to cancer medicines, resulting in death of the cancer cells.
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.
The medicine was not authorised anywhere in the EU for AML. Orphan designation of the medicine had been granted in the United States for AML.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 April 2017 recommending the granting of this designation.
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.
FGK Representative Service GmbH
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: