EU/3/10/758 - orphan designation for treatment of post-polycythaemia vera myelofibrosis

Pomalidomide
OrphanHuman

Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in November 2016 on request of the Sponsor.

On 27 July 2010, orphan designation (EU/3/10/758) was granted by the European Commission to Celgene Europe Limited, United Kingdom, for pomalidomide for the treatment of post-polycythaemia vera myelofibrosis.

Myelofibrosis is a disease in which the bone marrow (the spongy tissue inside the large bones) becomes dense and fibrous, and starts producing abnormal immature blood cells that replace the normal blood cells. It can develop as a reaction to polycythaemia vera (overproduction of red blood cells).

In myelofibrosis, some immature blood cells migrate from the bone marrow to other organs, such as the spleen and liver, where they mature. This causes the organs to become enlarged. Patients with the disease can develop several symptoms, including pain in the bones, fever, tiredness, weakness, weight loss, infections and bleeding.

Post-polycythaemia vera myelofibrosis is a debilitating disease that is long lasting and may be life threatening because it can lead to severe anaemia (low red blood cell counts) and infections, and can result in leukaemia (cancer of the white blood cells).

At the time of designation, post-polycythaemia vera myelofibrosis affected less than 0.01 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 500 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).

At the time of designation, although hydroxyurea and busulfan were authorised in the EU for primary myelofibrosis (myelofibrosis of unknown cause), there were no treatments authorised specifically for post-polycythaemia vera myelofibrosis.

Treatments for this disease were aimed at relieving symptoms. They included androgens (male hormones), glucocorticoids (a type of steroid) and erythropoietin (a hormone that stimulates the production of red blood cells) to treat anaemia, and surgery or radiation to remove or shrink the enlarged spleen. In some patients, bone marrow transplantation was used. This is a complex procedure where the bone marrow of the patient is destroyed and replaced with bone marrow from a matched donor.

Pomalidomide is an immunomodulating agent. This means that it affects the activity of the immune system. The way that pomalidomide will work in post-polycythaemia vera myelofibrosis is not known, but it is expected to block the growth of the abnormal cells in the bone marrow, while allowing normal cells to grow, including the cells that produce red blood cells. This is expected to improve anaemia in patients with this condition.

The effects of pomalidomide have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with pomalidomide in patients with myelofibrosis were ongoing.

At the time of submission, pomalidomide was not authorised anywhere in the EU for post-polycythaemia vera myelofibrosis 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 April 2010 recommending the granting of this designation.

  • 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

Active substance
Pomalidomide
Intended use
Treatment of post-polycythaemia vera myelofibrosis
Orphan designation status
Withdrawn
EU designation number
EU/3/10/758
Date of designation
Sponsor

Celgene Europe Limited
1 Longwalk Road
Stockley Park
Uxbridge
Middlesex UB11 1DB
United Kingdom
Telephone: +44 208 831 83 00
Telefax: +44 208 831 83 01
E-mail: medinfo.uk.ire@celgene.com

Review of designation

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in November 2016 on request of the Sponsor.

EMA list of opinions on orphan medicinal product designation

EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

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.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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