EU/3/10/831

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Orphan designation

On 23 February 2011, orphan designation (EU/3/10/831) was granted by the European Commission to Analytica International Inc, Germany, for autologous tumour-derived immunoglobulin idiotype coupled to keyhole limpet hemocyanin for the treatment of mantle cell lymphoma.

The sponsorship was transferred to Biovest Europe Limited, United Kingdom, in September 2011.

What is mantle cell lymphoma?

Mantle cell lymphoma is an aggressive cancer of a type of white blood cell called B-lymphocytes, or B cells. In mantle cell lymphoma, the B cells multiply too quickly and live for too long, so there are too many of them in the lymph nodes. The first sign of the disease is usually a lump in the neck, under the arm or in the groin area, caused by an enlarged lymph node. Patients may also have fever, weight loss, tiredness and night sweats.

Mantle cell lymphoma is usually diagnosed in people aged over 50 years. It is more common in men than women. Mantle cell lymphoma is a severe and life-threatening disease that is associated with poor overall survival.

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

At the time of designation, mantle cell lymphoma affected less than 0.3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 15,000 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. This represents a population of 506,500,000 (Eurostat 2010).

What treatments are available?

At the time of designation, temsirolimus was specifically authorised in the EU for the treatment of mantle cell lymphoma that has come back after previous treatment or has not responded to other treatments. The main treatments for mantle cell lymphoma included chemotherapy (medicines to treat cancer), immunotherapy (medicines that stimulate the body’s own immune system to kill the cancer cells) and radiotherapy (treatment with radiation). Haematopoietic (blood) stem cell transplantation was also used. This is a complex procedure where patients receive stem cells to help restore the bone marrow.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit to those affected by the condition because early studies show that it works in a different way to existing treatments, by inducing an immune response which may fight against the cancerous B cells. 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?

The medicine is a ‘cancer vaccine’. It is expected to work by activating the patient’s immune system (the body’s natural defences) so that it attacks and kills the cancer cells.

To prepare this medicine, cancer cells are extracted from a patient’s lymph node. A protein specific to the patient’s cancer cells is isolated from the cells, then attached to a carrier, ‘keyhole limpet haemocyanin’ (a protein prepared from a sea snail), to enhance the response of the body to the vaccine.

When this ‘patient-specific’ vaccine is given, the patient’s immune system is expected to recognise the cancer cell protein as ‘foreign’. This is expected to stimulate an immune response against the cancer cells, resulting in the immune system attacking them in the body.

What is the stage of development of this medicine?

The effects of autologous tumor-derived immunoglobulin idiotype coupled to keyhole limpet hemocyanin have been evaluated in experimental models.

At the time of submission of the application for orphan designation, one clinical trial in patients with mantle cell lymphoma had been carried out.

At the time of submission, this medicine was not authorised anywhere in the EU for the treatment of mantle cell lymphoma. Orphan designation of this medicine had been granted for the treatment of mantle cell lymphoma in the United States of America (USA), and for the treatment of follicular lymphoma in the EU and the USA.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 10 November 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.

Key facts

Product details for <p>Autologous tumour-derived immunoglobulin idiotype coupled to keyhole limpet hemocyanin</p>
Active substanceAutologous tumour-derived immunoglobulin idiotype coupled to keyhole limpet hemocyanin
Medicine Name
Disease/conditionTreatment of mantle cell lymphoma
Date of decision23/02/2011
OutcomePositive
Orphan decision numberEU/3/10/831

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor’s contact details:

Biovest Europe Limited
Evershed House
70 Great Bridgewater St.
Manchester M1 5ES
United Kingdom
Telephone: +44 845 497 8663
Telefax: +44 845 497 8888

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.