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Orphan designation

On 6 November 2006, orphan designation (EU/3/06/420) was granted by the European Commission to Wyeth Europa Limited, United Kingdom, for temsirolimus for the treatment of mantle cell lymphoma.


What is mantle cell lymphoma?

Mantle cell lymphoma belongs to the group of diseases called “non-Hodgkin lymphomas”. Lymphoma is a type of cancer that originates from the lymphatic system. The lymphatic system is part of the immune system: the body's natural defence against infection and disease. It is a complex system made up of organs such as the bone marrow (the spongy tissue inside the large bones in the body), the thymus and the spleen, and a network of lymph nodes throughout the body that are connected by lymphatic vessels. Normally, the lymphatic cells grow in a controlled manner. However, in lymphoma a lymphatic cell continues to grow and divides in an uncontrolled manner, developing into a tumour. Lymphoma cells generally grow in lymph nodes. There are several different types of lymphoma depending on the type of lymphatic cancer cells. In mantle cell lymphoma, the cancer cells are related to the blood cells called B-lymphocytes. Mantle cell lymphoma is a life-threatening condition.

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



What treatments are available?


Satisfactory argumentation has been submitted by the sponsor to justify the assumption that temsirolimus might be of potential significant benefit for the treatment of mantle cell lymphoma, mainly because it may work for patients where other treatments have failed. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Temsirolimus is designed to inhibit (block) the enzyme (a protein that triggers chemical reactions in the body) called serine/threonine kinase. This enzyme plays a role in a cascade of molecular reactions involved in the control of growth and division of cells. In cancer cells, the function of this enzyme is disturbed causing uncontrolled growth and too much division of the cancer cells, making them develop into tumours. According to the sponsor, temsirolimus might, by inhibition of the activity of serine/threonine kinase, help in slowing down or stopping the further growth of cancer cells.Describe as simply as possible the clinically relevant principal mechanism of action and try to link this to the condition applied for.

What is the stage of development of this medicine?

The effects of temsirolimus were evaluated in experimental models.


Temsirolimus was not authorised anywhere worldwide for the treatment of mantle cell lymphoma, at the time of submission. Orphan designation of temsirolimus was granted in the European Union for the treatment of renal cell carcinoma.


Update: temsirolimus (Torisel) has been authorised in the EU since 21 August 2009 for the treatment of adult patients with relapsed and/or refractory mantle cell lymphoma [MCL].

  • the seriousness of the condition;


Key facts

Product details for <p>Temsirolimus</p>
Active substanceTemsirolimus
Medicine NameTorisel
Disease/conditionTreatment of mantle cell lymphoma
Date of decision06/11/2006
Orphan decision numberEU/3/06/420

Review of designation

Sponsor’s contact details:

Pfizer Limited
Ramsgate Road
Kent CT13 9NJ
United Kingdom
Telephone: +44 1304 61 61 61
Telefax: +44 1304 65 21 44

Patients' organisations:

