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Orphan designation

On 26 November 2009, orphan designation (EU/3/09/694) was granted by the European Commission to Only For Children Pharmaceuticals, France, for 6-thioguanine (oral liquid) for the treatment of acute lymphoblastic leukaemia.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What is acute lymphoblastic leukaemia?

Acute lymphoblastic leukaemia (ALL) is a cancer of the white blood cells called lymphocytes. In this disease, the lymphocytes multiply too quickly and live for too long, so there are too many of them circulating in the blood. These abnormal lymphocytes are not fully developed and do not work properly. Over a period of time, they replace the normal white cells and red blood cells and platelets in the bone marrow (the spongy tissue inside the large bones in the body).

ALL is the most common type of leukaemia in young children, but the disease also affects adults, especially those aged 65 years and older. Many people with ALL can be cured. However, despite the available treatments, ALL remains a serious and life-threatening disease in some patients.

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

At the time of designation, ALL affected less than 1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 50,000 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 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 504,800,000 (Eurostat 2009).

What treatments are available?

Treatment for ALL 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 treatment of ALL was chemotherapy (medicines to treat cancer) followed by or combined with radiotherapy (treatment with radiations). Bone marrow transplantation was also used. This is a complex procedure where the bone marrow of the patient is destroyed and replaced with healthy bone marrow from a matched donor.

The sponsor has provided sufficient information to show that 6-thioguanine (oral liquid) might be of significant benefit for patients with ALL because of the way the medicine is given. 6-Thioguanine is already available in the EU as tablets for the treatment of ALL. 6-Thioguanine (oral liquid) is a liquid form of 6-thioguanine, which could be given to children who have difficulty swallowing tablets. This may make it easier for children to take the medicine and allow the dose to be adjusted more precisely according to the patient’s weight. 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?

6-Thioguanine (oral liquid) is expected to work in the same way as 6-thioguanine tablets. 6-Thioguanine is an anticancer medicine that belongs to the group ‘antimetabolites’. It works by interfering with the production of DNA within cells, preventing them from making more copies of their DNA. This means that cancer cells cannot divide and they eventually die.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of the effects of 6-thioguanine (oral liquid) in experimental models was planned.

At the time of submission of the application for orphan designation, no clinical trials with the designated product in patients with ALL had been started.

At the time of submission, 6-thioguanine (oral liquid) was not authorised anywhere in the EU for ALL or designated as orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 October 2009 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 Community) 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.

Key facts

Product details for <p>6-Thioguanine (oral liquid)</p>
Active substance6-Thioguanine (oral liquid)
Medicine Name
Disease/conditionTreatment of acute lymphoblastic leukaemia
Date of decision25/11/2009
Orphan decision numberEU/3/09/694

Review of designation

Sponsor’s contact details

Only For Children Pharmaceuticals
Site Villemin, 10
Avenue de Verdun
75010 Paris
Tel. +33 1 57 27 86 86
Fax +33 1 77 65 63 94

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.