EU/3/08/555

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Orphan designation

On 8 July 2008, orphan designation (EU/3/08/555) was granted by the European Commission to QuadraMed Limited, United Kingdom, for vincristine sulphate liposomes for the treatment of acute lymphoblastic leukaemia.

The sponsorship was transferred to Fulcrum Pharma (Europe) Limited, United Kingdom, in April 2009 and subsequently to NDA Regulatory Science Ltd, United Kingdom, in May 2011.

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 leukaemic lymphocytes look normal, but they are not fully developed and do not work properly. Over a period of time, these abnormal cells replace the normal white and red blood cells and platelets in the bone marrow (the spongy tissue inside the large bones in the body that produces blood cells). ALL is the most common type of leukaemia in young children but the disease also affects adults, especially those aged 65 and older. Many people with acute leukaemia can be cured. However, despite the available treatments, ALL remains a serious and life-threatening condition in some patients.

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

At the time of designation, ALL affected approximately 0.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 25,000 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 based on data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 502,282,000 (Eurostat 2008).

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. The main treatment is chemotherapy (medicines used to kill cancer cells) followed by or combined with radiotherapy (using radiation to kill cancer cells). Bone marrow transplantation is also used.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that vincristine sulphate liposomes might be of potential significant benefit for the treatment of ALL because it has a new delivery method. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain orphan status.

How is this medicine expected to work?

Vincristine is a medicine that is already used widely as chemotherapy for the treatment of blood cancers including ALL. It is a ‘vinca alkaloid’ (a substance obtained from the Vinca periwinkle flower) that blocks the cell's ability to break down the ‘skeleton’ that allows cells to divide and multiply. With the skeleton still in place the cells cannot divide, and they eventually die. In this medicine, vincristine is contained in liposomes (tiny fatty particles). This is expected to improve the way the medicine works compared with the conventional form of the medicine, by slowing down the clearance of the medicine from the body.

What is the stage of development of this medicine?

The effects of vincristine sulphate liposomes have been evaluated in experimental models.

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

Vincristine sulphate liposomes were not authorised anywhere worldwide for ALL at the time of submission but orphan designation of vincristine sulphate liposomes has been granted in the United States of America for the treatment of ALL.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 14 May 2008 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;
  • and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

Key facts

Product details for <p>Vincristine sulphate liposomes</p>
Active substanceVincristine sulphate liposomes
Medicine Name
Disease/conditionTreatment of acute lymphoblastic leukaemia
Date of decision08/07/2008
OutcomePositive
Orphan decision numberEU/3/08/555

Review of designation

Sponsor’s contact details:

NDA Regulatory Science Ltd
Prime House
Challenge Court
Barnett Wood Lane
Leatherhead
Surrey KT22 7DE
United Kingdom
Telephone: +44 1372 860 610
Telefax: +44 1372 860 611
E-mail: london@ndareg.com

Patients’ associations contact points:

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.