EU/3/09/672

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Orphan designation

On 8 October 2009, orphan designation (EU/3/09/672) was granted by the European Commission to Celgene Europe Limited, United Kingdom, for pomalidomide 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 life-threatening disease that leads to poor long-term survival.

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

At the time of designation, multiple myeloma affected approximately 2.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 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 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 504,800,000 (Eurostat 2009).

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 pomalidomide might be of significant benefit for patients with multiple myeloma because it might be used in patients who do not respond to existing treatments. In addition, the medicine will be available as capsules, whereas some 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?

Pomalidomide is an immunomodulating agent. This means that it affects the activity of the immune system. Pomalidomide is expected to work in a number of ways in multiple myeloma, in a similar way to other immunomodulating agents such as lenalidomide and thalidomide: it is expected to block the development of tumour cells, by preventing the growth of blood vessels within tumours and thereby reducing the supply of oxygen and nutrients to the cancer cells. It is also expected to stimulate some of the specialised cells of the immune system to attack the cancerous cells.

What is the stage of development of this medicine?

The effects of pomalidomide have been evaluated in experimental models.

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

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

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 July 2009 recommending the granting of this designation.

Update: Pomalidomide (Imnovid, previously Pomalidomide Celgene) has been authorised in the EU since 5 August 2013. Imnovid in combination with dexamethasone is indicated in the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least two prior treatment regimens, including both lenalidomide and bortezomib, and have demonstrated disease progression on the last therapy.

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.

Name Language First published Last updated
EU/3/09/672: Public summary of opinion on orphan designation for pomalidomide for the treatment of multiple myeloma (English only) 20/10/2009 16/10/2014

Key facts

Product details for <p>Pomalidomide</p>
Active substancePomalidomide
Medicine NamePomalidomide Celgene
Disease/conditionTreatment of multiple myeloma
Date of decision08/10/2009
OutcomePositive
Orphan decision numberEU/3/09/672

Review of designation

During its meeting of 11-13 June 2013, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/09/672 for Imnovid, previously Pomalidomide Celgene, (pomalidomide) as an orphan medicinal product for the treatment of multiple myeloma. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other satisfactory methods of treatment. As other methods of treatment for patients with this condition are authorised in the European Union (EU), the COMP also looked at the significant benefit of the product over existing treatments. The COMP recommended that the orphan designation of the medicine be maintained*.


*The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with a comparable therapeutic indication cannot be placed on the market.

For a list of the administrative updates please refer to the PDF document below.

Life-threatening or long-term debilitating nature of the condition

The Committee for Medicinal Products for Human Use (CHMP) recommended the authorisation of Imnovid:

‘in combination with dexamethasone for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least two prior treatment regimens, including both lenalidomide and bortezomib, and have demonstrated disease progression on the last therapy’.

This falls within the scope of the product’s designated orphan indication, which is ‘treatment of multiple myeloma’.

The COMP concluded that there had been no change in the seriousness of the condition since the orphan designation in 2009. Multiple myeloma remains a debilitating and life-threatening disease because it disrupts the normal functioning of the bone marrow, leads to bone destruction and causes kidney failure.

Prevalence of the condition

The sponsor provided updated information on the prevalence of multiple myeloma based on data from the Globocan 2008 database.

On the basis of the information provided by the sponsor and the knowledge of the COMP, the COMP concluded that the prevalence of multiple myeloma remains below the ceiling for orphan designation, which is 5 people in 10,000. At the time of the review of the orphan designation, the prevalence was estimated to be approximately 1.3 people in 10,000. This is equivalent to a total of around 66,000 people in the EU.

Existence of other satisfactory methods of treatment

At the time of the review of the orphan designation, several medicines were authorised for multiple myeloma in the EU, including bortezomib, lenalidomide and thalidomide. They were usually combined with steroids to reduce the activity of the immune system, the body’s natural defences. Where these medicines did not work, some patients received an allogeneic stem-cell transplant (a complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow).

Significant benefit over existing treatments

The COMP concluded that the claim of a significant benefit of Imnovid in multiple myeloma is justified because of its demonstrated effectiveness in patients whose disease did not respond to or had come back after previous treatment, including bortezomib and lenalidomide. These patients have very limited treatment options.

The COMP conclusions are supported by data from 3 studies in this group of patients, which showed that Imnovid was effective at delaying the progression of multiple myeloma.

Therefore, although other satisfactory methods for the treatment of multiple myeloma have been authorised in the EU, the COMP concluded that Imnovid is of significant benefit for patients with this condition.

Conclusions

Based on the data submitted and the scientific discussion within the COMP, the COMP considered that Imnovid still meets the criteria for designation as an orphan medicinal product and that it should remain in the Community Register of Orphan Medicinal Products.

Further information on the current regulatory status of Pomalidomide Celgene can be found in the European public assessment report (EPAR).

Sponsor’s contact details

Celgene Europe Limited
1 Longwalk Road
Stockley Park
Uxbridge
Middlesex
UB11 1DB
United Kingdom
Tel. +44 (0)20 8831 8300
Fax +44 (0)20 8831 8301
E-mail: medinfo.uk.ire@celgene.com

Patient 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.