EU/3/08/538

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Orphan designation

On 2 April 2008, orphan designation (EU/3/08/538) was granted by the European Commission to Pharmion Ltd, United Kingdom, for amrubicin hydrochloride for the treatment of small-cell lung cancer.

The sponsorship was transferred to Celgene Europe Limited, United Kingdom, in December 2008.

What is small-cell lung cancer?

Small-cell lung cancer is a disease in which cancer cells develop in the lungs. This type of lung cancer accounts only for about 20% of all lung cancer cases. Usually it develops in the central part of the lungs, and the cancer cells are typically small compared with the other types of lung cancer. Signs of cancer are difficult to detect in early stages of the disease, and the majority of the patients are diagnosed when the disease has spread and cannot be surgically removed. Small-cell lung cancer is a life-threatening disease.

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

At the time of designation, small-cell lung cancer affected less than 1.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 75,000 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. This represents a population of 498,000,000 (Eurostat 2006).

What treatments are available?

The main treatment of small-cell lung cancer consists of chemotherapy (i.e drugs that kill cancer cells) and radiotherapy (i.e using high-dose x-rays or other high-energy rays to kill cancer cells).

Several medicinal products were authorised in the European Community, for the treatment of small-cell lung cancer, at the time of submission of the application for orphan designation. Amrubicin hydrochloride might be of potential significant benefit for the treatment of small-cell lung cancer, because it might improve the long-term outcome of the 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?

Amrubicin hydrochloride is a synthetic anthracycline, a type of chemotherapy. Within the tumour, amrubicin hydrochloride is transformed into a substance called amrubicinol. Amrubicin hydrochloride and amrubicinol both stop the production of new genetic material and can therefore kill dividing cells. As tumour cells divide very rapidly, the product is thought to decrease tumour growth through its action on dividing cells.

What is the stage of development of this medicine?

The effects of amrubicin hydrochloride were evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials in patients with small-cell lung cancer were ongoing and some were completed.

Amrubicin hydrochloride was authorised in Japan for the treatment of both small-cell lung cancer and non-small cell lung cancer, at the time of submission.

Orphan designation of amrubicin hydrochloride was not granted anywhere worldwide for small-cell lung cancer.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 February 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;
  • 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>Amrubicin hydrochloride</p>
Active substanceAmrubicin hydrochloride
Medicine Name
Disease/conditionTreatment of small cell lung cancer
Date of decision02/04/2008
OutcomePositive
Orphan decision numberEU/3/08/538

Review of designation

Sponsor’s contact details:

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

Patients’ associations contact points:

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.