EU/3/08/538: Orphan designation for the treatment of 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.

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).

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.

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.

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.

• 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.