EU/3/10/792

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Orphan designation

On 1 October 2010, orphan designation (EU/3/10/792) was granted by the European Commission to ImmunoGen Europe Limited, United Kingdom, for maytansinoid-conjugated humanised monoclonal antibody against CD56 for the treatment of small-cell lung cancer.

What is small-cell lung cancer?

Small-cell lung cancer is a type of lung cancer that usually develops in the central part of the lungs, and in which the cancer cells are small compared with other types of lung cancer. Small-cell lung cancer is almost always caused by smoking. The cancer is difficult to detect in the early stages of the disease, and the majority of the patients are diagnosed when the cancer has spread and cannot be removed by surgery.

Small-cell lung cancer is a life-threatening disease that is associated with poor long-term survival.

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

At the time of designation, small-cell lung cancer affected approximately 1 in 10,000 people in the European Union (EU). This was 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. At the time of designation, this represented a population of 506,300,000 (Eurostat 2010).

What treatments are available?

At the time of designation, several medicines were authorised in the EU for the treatment of small-cell lung cancer. The choice of treatment depended mainly on how advanced the disease was. Treatments included chemotherapy (medicines to treat cancer) and radiotherapy (treatment with radiation).

The sponsor has provided sufficient information to show that maytansinoid-conjugated humanised monoclonal antibody against CD56 might be of significant benefit for patients with small-cell lung cancer because it works in a different way to existing treatments and because early studies show that it might be used in combination with existing treatments to improve the outcome of patients with this condition. 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?

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 small-cell-lung-cancer 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 lung-cancer cells. The antibody-maytansinoid complex then enters the cancerous cells, where the maytansinoid is released to exert its anti-tumour effect by blocking cell division, leading to cell death. This is expected to slow down the growth or cause the shrinkage of small-cell-lung-cancer tumours.

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 small-cell lung cancer were ongoing.

At the time of submission, this medicine was not authorised anywhere in the EU for small-cell lung cancer 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 8 July 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 small-cell lung cancer
Date of decision01/10/2010
OutcomePositive
Orphan decision numberEU/3/10/792

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

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.