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Orphan designation

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in January 2014 on request of the Sponsor.

On 10 June 2010, orphan designation (EU/3/10/740) was granted by the European Commission to Æterna Zentaris GmbH, Germany, for perifosine for the treatment of multiple myeloma.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

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 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 less than 2.2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 111,000 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. This represents a population of 506,500,000 (Eurostat 2010).

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 perifosine 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 patients who do not respond to existing treatments. In addition, perifosine will be available as tablets, whereas most of the existing treatments need to be given by injection. 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?

Perifosine is expected to work by blocking some enzymes, known as protein kinases B (or Akt). These enzymes are involved in stimulating cells to grow and divide. Cancerous cells grow and divide uncontrollably. By blocking these enzymes, the medicine is expected to help control the growth and division of the cancerous cells and slow down the development of multiple myeloma.

What is the stage of development of this medicine?

The effects of perifosine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with perifosine in patients with multiple myeloma were ongoing.

At the time of submission, perifosine was not authorised anywhere in the EU for multiple myeloma. Orphan designation of perifosine had been granted in the United States of America for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 3 February 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.

Name Language First published Last updated
EU/3/10/740: Public summary of opinion on orphan designation Perifosine for the treatment of multiple myeloma (English only) 2010-06-24 2014-02-07

Key facts

Product details for <p>Perifosine</p>
Active substancePerifosine
Medicine Name
Disease/conditionTreatment of multiple myeloma
Date of decision10/06/2010
Orphan decision numberEU/3/10/740

Review of designation

Sponsor’s contact details

Æterna Zentaris GmbH
Weismuellerstr. 50
60314 Frankfurt Main
Tel. + 49 6942 6023475
Fax + 49 6942 6023404

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.