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Orphan designation

On 10 June 2010, orphan designation (EU/3/10/741) was granted by the European Commission to Choice Pharma Limited, United Kingdom, for pralatrexate for the treatment of cutaneous T-cell lymphoma.

The sponsorship was transferred to Allos Therapeutics Limited, United Kingdom, in September 2010.

What is cutaneous T-cell lymphoma?

Cutaneous T-cell lymphoma (CTCL) is a cancer of the lymphatic system, a network of vessels that transport fluid from tissues through the lymph nodes and into the bloodstream. In CTCL there is uncontrolled growth of the T-lymphocytes (T-cells), a type of white blood cell found in the lymphatic system. The cancerous T-cells appear in the skin, causing lesions (rashes, plaques and tumours) which can be itchy and painful.

CTCL usually happens in people aged between 40 and 60 years. In many cases, the disease is long-lasting, with survival for more than 10 to 20 years being common. However, it can be a serious and life-threatening disease because it can develop into more aggressive forms of cancer. The condition may have a large impact on quality of life, particularly because the skin lesions can cause disfigurement.

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

At the time of designation, CTCL affected approximately 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 51,000 people, and is below the threshold 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. This represents a population of 506,500,000 (Eurostat 2010).

What treatments are available?

At the time of designation, several products were authorised for the treatment of CTCL within the EU. Treatments for CTCL can be divided into topical (affecting the skin only) and systemic (affecting the whole body):

  • topical treatments are medicines applied to the skin including corticosteroids (a group of steroid medicines that reduce the division of T-cells), ultraviolet light and X-rays;
  • systemic treatments include cytotoxic medicines (medicines that kill cells that are dividing, such as cancer cells), interferon alfa (a medicine that helps the immune system to fight against the cancer cells) and photopheresis. Photopheresis is a technique in which blood is temporarily removed from the body to be treated with ultraviolet light. A substance is first added to the blood, that, when exposed to ultraviolet light, becomes activated and able to damage the T-cells. When these damaged cells are re-introduced in the patient’s blood, they trigger the immune system to attack and kill cancerous T-cells in the body.

The sponsor has provided sufficient information to show that pralatrexate might be of significant benefit for patients with CTCL because early studies show that it might improve the treatment of patients with this condition, particularly patients who are not responding to existing treatments. 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?

Pralatrexate is an ‘antimetabolite’ medicine. In the body, it is expected to take the place of folic acid and attach to an enzyme called dihydrofolate reductase (DHFR). DHFR is necessary for the production of new DNA and proteins, which are required for cells to divide and multiply. By attaching to DHFR, pralatrexate is expected to block the enzyme’s activity, inhibiting the growth of the cancer cells and eventually killing them.

What is the stage of development of this medicine?

The effects of pralatrexate have been evaluated in experimental models.

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

At the time of submission, pralatrexate was not authorised anywhere in the EU for CTCL. Orphan designation of pralatrexate had been granted in the United States of America for T-cell lymphoma.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 3 February 2010 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.

Name Language First published Last updated
EU/3/10/741: Public summary of opinion on orphan designation: Pralatrexate for the treatment of cutaneous T-cell lymphoma (English only) 2010-06-24 2011-03-10

Key facts

Product details for <p>Pralatrexate</p>
Active substancePralatrexate
Medicine Name
Disease/conditionTreatment of cutaneous T-cell lymphoma
Date of decision10/06/2010
Orphan decision numberEU/3/10/741

Review of designation

Sponsor’s contact details

Allos Therapeutics Limited
71 Knowl Piece
Wilbury Way
Hertfordshire SG4 OTY
United Kingdom
Telephone: +44 1462 424 416
Telefax: +44 1462 600 453

Patient 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.