EU/3/01/068

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Orphan designation

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in December 2008 on request of the Sponsor.

On 19 December 2001, orphan designation (EU/3/01/068) was granted by the European Commission to Kendle International Limited, United Kingdom, for thalidomide for the treatment of multiple myeloma.

What is multiple myeloma?

Multiple myeloma is a cancer of a type of white blood cell called a plasma cell. Plasma cells are found in the bone marrow. The bone marrow is the spongy tissue inside the large bones in the body. Normally, the bone marrow makes cells called “blasts” that mature into several different types of blood cells that have specific functions in the body. These include red cells, white cells and platelets. Red blood cells carry oxygen and other materials to all tissues of the body. Platelets make the blood clot, and white blood cells fight infection. In multiple myeloma an excessive number of plasma cells are produced. Normally, the division of cells takes place in a controlled manner but with multiple myeloma, the process gets out of control and abnormal plasma cells multiply, producing many myeloma cells. These fill up the bone marrow and interfere with production of the normal white cells, red cells and platelets. This leads to a number of possible complications, which include anaemia, bone pain and fractures, raised levels of calcium in the blood and kidney disease. Multiple myeloma is life-threatening.

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

At the time of designation, multiple myeloma affected approximately 1.2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 45,000 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 knowledge of the Committee for Orphan Medicinal Products (COMP).

*Disclaimer: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 377,000,000 (Eurostat 2001) and may differ from the true number of patients affected by the condition.

What treatments are available?

The main treatment of multiple myeloma is chemotherapy (using drugs to kill cancer cells) usually combined with steroids (a group of chemical substances, belonging to a larger class of molecules, the so-called hormones, which have an effect on the activity of certain organs). Several products were authorised for the condition in the Community at the time of submission of the application for orphan drug designation. Radiotherapy (using hig-dose x-rays or other hig-energy rays to kill cancer cells) can be very useful to treat painful areas and weakened bones. Interferon alpha is a protein normally produced by the body during viral infections, such as flu. Sometimes a combination of interferon and chemotherapy may be used.
Thalidomide could be of potential significant benefit for the treatment of multiple myeloma. The main reason is that it may offer a new way of killing cancer cells and stopping tumour growth in these patients. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Cancers need to produce a network of new blood vessels in order to grow. Without forming these blood vessels cancers cannot grow. The theory is that thalidomide will prevent the myeloma from growing by preventing the development of new blood vessels and possibly also reducing the supply of oxygen and nutrients to the cancer cells.

What is the stage of development of this medicine?

The evaluation of the effects of thalidomide in experimental models is ongoing.
At the time of submission of the application for orphan designation, clinical trials in patients with multiple myeloma were ongoing.

Thalidomide was not marketed anywhere worldwide for multiple myeloma, at the time of submission. Orphan designation of thalidomide was granted in the United States for the treatment of multiple myeloma.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 7 September 2001 a positive opinion recommending the grant of the above-mentioned 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 Community) 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>Thalidomide</p>
Active substanceThalidomide
Medicine Name
Disease/conditionTreatment of multiple myeloma
Date of decision18/12/2001
OutcomeWithdrawn
Orphan decision numberEU/3/01/068

Review of designation

Sponsor’s contact details:

Kendle International Ltd
Angel House
24 Station Road
Ely, Cambs CB7 4BS
United Kingdom
Telephone: +44 13 53 66 97 89
Telefax: +44 13 53 66 84 73
E-mail: info.ely@kendle.com

Patients’ associations contact points:

International Myeloma Foundation (UK)
Lower Ground Floor
37 York Place
Edinburgh
Scotland
EH1 3HP
United Kingdom
Myeloma Infoline: 0800 980 3332 (freephone for UK)
Telephone: +44 13 15 57 33 32 (Administration)
Telefax: +44 13 15 56 97 20
E-mail: TheIMF@myeloma.org.uk

Austrian Myeloma Association
Bürglsteinstr. 21-10
5020 Salzburg
Austria

Danish Myeloma Association
(Dansk Myelomatose Forening)
v/ Peter Randløv
Klosterbakken 40
DK-3500 Værløse
Denmark
Telephone: +45 44 48 41 27
E-mail: randlov@post9.tele.dk