EU/3/08/593

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Orphan designation

On 3 December 2008, orphan designation (EU/3/08/593) was granted by the European Commission to Biotest AG, Germany, for N2'-Deacetyl-N2'-[4-methyl-4-(oxobuthyldithio)-1-oxopentyl]-maytansine-chimerized anti-CD138 IgG4 monoclonal antibody 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 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 life-threatening disease.

What is the estimated number of patients affected by multiple myeloma?

At the time of designation, multiple myeloma affected approximately 2.2 in 10,000 people in the European Union (EU)*. This is below the threshold for orphan designation which is 5 in 10,000 people, and is equivalent to a total of around 111,000 people. 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 based on data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 502,282,000 (Eurostat 2008).

What treatments are available?

At the time of submission of the application for orphan designation, several medicines were already authorised for multiple myeloma in the EU. The main treatment for multiple myeloma is chemotherapy (medicines to treat cancer) usually combined with steroids (medicines used to reduce the activity of the immune system). Treatment with radiotherapy (using radiation to kill cancer cells) can be very useful in treating pain and weakened bones. Interferon alfa, a protein normally produced by the body during viral infections, can sometimes be used in combination with chemotherapy.

N2'-Deacetyl-N2'-[4-methyl-4-(oxobuthyldithio)-1-oxopentyl]-maytansine-chimerized anti-CD138 IgG4 monoclonal antibody could be of potential significant benefit for the treatment of multiple myeloma because it works in a different way to other medicines and could be more selective for multiple myeloma. 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?

N2'-Deacetyl-N2'-[4-methyl-4-(oxobuthyldithio)-1-oxopentyl]-maytansine-chimerized anti-CD138 IgG4 monoclonal antibody is made up of two components:

  • a monoclonal antibody (a type of protein) that has been designed to recognise and bind to a specific structure called CD138. CD138 is a protein that is found on the surface of multiple myeloma cells, which helps the cells stick to each other and to the extracellular matrix (the framework of fibres surrounding the cells that provides support for their growth) as they enter the bone marrow;
  • maytansine, a substance that kills cancer cells by interfering with their division.

The medicine is expected to bind to multiple myeloma cells via its monoclonal antibody part. Once on the cells, maytansine is expected to act locally and stop any further cell division. Through this mechanism, the medicine might lead to the death of multiple myeloma cells.

What is the stage of development of this medicine?

The effects of N2'-deacetyl-N2'-[4-methyl-4-(oxobuthyldithio)-1-oxopentyl]-maytansine-chimerized anti-CD138 IgG4 monoclonal antibody have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with multiple myeloma had been started.

At the time of submission, N2'-deacetyl-N2'-[4-methyl-4-(oxobuthyldithio)-1-oxopentyl]-maytansine-chimerized anti-CD138 IgG4 monoclonal antibody was not authorised anywhere in the world for multiple myeloma or designated as 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 October 2008 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;
  • and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

Key facts

Product details for <p>N2'-Deacetyl-N2'-[4-methyl-4-(oxobuthyldithio)-1-oxopentyl]-maytansine-chimerized anti-CD138 IgG4 monoclonal antibody</p>
Active substanceN2'-Deacetyl-N2'-[4-methyl-4-(oxobuthyldithio)-1-oxopentyl]-maytansine-chimerized anti-CD138 IgG4 monoclonal antibody
Medicine Name
Disease/conditionTreatment of multiple myeloma
Date of decision02/12/2008
OutcomePositive
Orphan decision numberEU/3/08/593

Review of designation

Sponsor’s contact details:

Biotest AG
Landsteinerstrasse 5
D-63303 Dreieich
Germany
Telephone: +49 61 03 80 10
Telefax: +49 61 03 80 11 80
E-mail: sibylle_kaiser@biotest.de

Patients’ associations contact points:

Ligue Nationale Contre le Cancer
14 Rue Corvisart
75013 Paris
France
Telephone: +33 1 53 55 24 00
Telefax: +33 1 43 36 91 10
E-mail: ligue@ligue-cancer.net

Macmillan Cancer Support (merged with CancerBACUP)
3 Bath Place
Rivington Street
London
EC2A 3JR
United Kingdom
Telephone: +44 20 7696 9003
Switchboard open during office hours, Mon–Fri, 9am–Noon and 2pm–4.45pm
Telefax: +44 20 7696 9002

Deutsche Krebshilfe e.V.
Buschstr. 32
53113 Bonn
Germany
Telephone: +49 2 287 29 900
Telefax: +49 2 287 29 90 11
E-mail: deutsche@krebshilfe.de