EU/3/10/746

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Orphan designation

On 9 June 2010, orphan designation (EU/3/10/746) was granted by the European Commission to ImmunoGen Europe Limited, United Kingdom, for maytansinoid-conjugated humanised monoclonal antibody against CD56 for the treatment of Merkel-cell carcinoma.

What is Merkel-cell carcinoma?

Merkel-cell carcinoma (also known as cutaneous neuro-endocrine carcinoma) is a type of skin cancer that starts in ‘neuro-endocrine’ cells called Merkel cells. Neuro-endocrine cells release hormones into the blood when stimulated by the nervous system. Merkel-cell carcinoma usually appears as a painless red-blue lump on the head and neck, but it can also be found on the arms and legs, and sometimes on the trunk.

Merkel-cell carcinoma is different from other skin cancers in that it grows rapidly. The disease is life-threatening because it starts to spread quickly to other parts of the body. Once this cancer has spread, it is associated with very poor long-term survival.

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

At the time of designation, Merkel-cell carcinoma affected approximately 0.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 20,000 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. At the time of designation, this represented a population of 506,300,000 (Eurostat 2010).

What treatments are available?

At the time of designation, no satisfactory methods were authorised in the EU to treat Merkel-cell carcinoma. Patients were treated with chemotherapy (medicines to treat cancer), surgery and radiotherapy (treatment with radiation). However, in most patients whose disease had spread, the disease tended to come back after treatment.

How is this medicine expected to work?

Maytansinoid-conjugated humanised monoclonal antibody against CD56 is made up of two parts:

  • a monoclonal antibody against CD56, a type of protein that has been designed to recognise and attach to a specific structure (antigen) called CD56. CD56 is a receptor that is found at high levels on the surface of cancer cells of neuro-endocrine origin, including Merkel cells;
  • a maytansinoid called DM1. This is a ‘cytotoxic’ substance that kills cancer cells when they attempt to divide.

The antibody part allows the medicine to attach to CD56 on the surface of cancerous Merkel cells.

The antibody-maytansinoid complex then enters the cancerous cells, where the maytansinoid exerts its anti-tumour effect by blocking cell division. This is expected to slow down the growth of Merkel-cell carcinoma.

What is the stage of development of this medicine?

The effects of maytansinoid-conjugated humanised monoclonal antibody against CD56 have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with this medicine in patients with Merkel -ell carcinoma were ongoing.

At the time of submission, maytansinoid-conjugated humanised monoclonal antibody against CD56 was not authorised anywhere in the EU for Merkel-cell carcinoma or designated as an 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 3 March 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>Maytansinoid-conjugated humanised monoclonal antibody against CD56</p>
Active substanceMaytansinoid-conjugated humanised monoclonal antibody against CD56
Medicine Name
Disease/conditionTreatment of Merkel-cell carcinoma
Date of decision09/06/2010
OutcomePositive
Orphan decision numberEU/3/10/746

Review of designation

Sponsor’s contact details

ImmunoGen Europe Limited
c/o Reeves LLC
24 Chiswell Street
Third Floor
London
EC1Y 4YX
United Kingdom
Tel. +44 (0)20 7382 1820
Fax +44 (0)20 7382 1821
E-mail: clinicaltrials@immunogen.com

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.