EU/3/04/255 - orphan designation for treatment of small cell lung cancer

Sabarubicin
OrphanHuman

Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in March 2009 on request of the Sponsor.

On 21 December 2004, orphan designation (EU/3/04/255) was granted by the European Commission to Menarini Ricerche S.p.A , Italy, for sabarubicin for the treatment of small cell lung cancer.

Small cell lung cancer is a disease in which cancer (malignant) 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 disseminated and cannot be surgically removed. Small cell lung cancer is a life-threatening disease.

At the time of designation, small cell lung cancer affected approximately 0.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 23,000 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 based on data from the European Union (EU 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004).

The main treatment of small cell lung cancer is 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 for the treatment of small cell lung cancer, alone or in combination in the Community at the time of submission of the application for orphan designation. Sabarubicin might be of potential significant benefit for the treatment of small cell lung cancer because it might improve the long-term outcome. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

Sabarubicin is expected to kill dividing cells since it might interfere with the production of a structure called deoxyribonucleic acid (DNA), the cellular structure which carries the genetic information and which is necessary for the division and survival of both normal and abnormal (i.e. tumour) cells.

The effects of sabarubicin 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.
Sabarubicin was not marketed anywhere worldwide for small cell lung cancer or designated as orphan medicinal product elsewhere for this condition, at the time of submission.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 11 November 2004 a positive opinion recommending the grant of the above-mentioned 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 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

Active substance
Sabarubicin
Intended use
Treatment of small cell lung cancer
Orphan designation status
Withdrawn
EU designation number
EU/3/04/255
Date of designation
Sponsor

Menarini Ricerche S.p.A
Via Tito Speri 10
I-00040 Pomezia (Roma)
Italy
Telephone: +39 055 568 03 13
Telefax: +39 055 56 80 99 54
E-mail: sabarubicin@menarini-ricerche.it

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:

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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