EU/3/10/835

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Orphan designation

On 23 February 2011, orphan designation (EU/3/10/835) was granted by the European Commission to ImmunoGen Europe Limited, United Kingdom, for maytansinoid-conjugated humanised monoclonal antibody against CD56 for the treatment of multiple myeloma.

What is multiple myeloma?

Multiple myeloma is a cancer of a type of white blood cell called plasma cells. Plasma cells in multiple myeloma are found in the bone marrow, the spongy tissue inside the large bones in the body. In multiple myeloma, the division of plasma cells becomes out of control, resulting in abnormal, immature plasma cells multiplying and filling up the bone marrow. This interferes with production of normal white blood cells, red blood cells and platelets (components that help the blood to clot), leading to complications such as anaemia (low red-blood-cell counts), bone pain and fractures, raised blood calcium levels and kidney disease.

Multiple myeloma is a debilitating and 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, multiple myeloma affected approximately 2.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 127,000 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. At the time of designation, this represented a population of 507,700,000 (Eurostat 2011).

What treatments are available?

At the time of designation, several medicines were authorised for multiple myeloma in the EU. The main treatment for multiple myeloma was chemotherapy (medicines to treat cancer) usually combined with steroids to reduce the activity of the immune system, the body’s natural defences. Radiotherapy (treatment with radiation) was considered to be very useful in treating pain and weakened bones. Interferon alfa, a protein normally produced by the body during viral infections, was sometimes used in combination with chemotherapy.

The sponsor has provided sufficient information to show that maytansinoid-conjugated humanised monoclonal antibody against CD56 might be of significant benefit for patients with multiple myeloma because it works in a different way to existing treatments, and early studies show that it might be used in combination with other treatments to improve the outcome of patients with this condition. These assumptions 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, 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 myeloma cells;
  • a maytansinoid called DM1, a ‘cytotoxic’ substance that kills cells when they attempt to divide.

The antibody part allows the medicine to attach to CD56 on the surface of cancer cells. The antibody-maytansinoid complex then enters the cancerous cells, where the maytansinoid is released to exert its cytotoxic effect by blocking cell division, leading to cell death. This is expected to slow down the growth or cause the shrinkage of multiple myeloma 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 the medicine in patients with multiple myeloma and other types of cancer were ongoing.

At the time of submission, this medicine was not authorised anywhere in the EU for multiple myeloma. Orphan designation of maytansinoid-conjugated humanised monoclonal antibody against CD56 had been granted in the EU and the United States for Merkel-cell carcinoma and small-cell lung carcinoma.

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>Maytansinoid-conjugated humanised monoclonal antibody against CD56</p>
Active substanceMaytansinoid-conjugated humanised monoclonal antibody against CD56
Medicine Name
Disease/conditionTreatment of multiple myeloma
Date of decision23/02/2011
OutcomePositive
Orphan decision numberEU/3/10/835

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 18 21
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.