EU/3/09/696: Orphan designation for the treatment of ovarian cancer

Ovarian cancer is cancer of the ovaries (two organs in the female reproductive system that produce eggs). Most ovarian cancers occur in women over the age of 50 years. Due to the absence of symptoms in the early stages of the disease, the majority of patients are diagnosed when the cancer has started to spread to other parts of the body.

Ovarian cancer is a life-threatening disease that is associated with poor long-term survival.

At the time of designation, ovarian cancer affected approximately 3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 151,000 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).

At the time of designation, several medicines were authorised in the EU for the treatment of ovarian cancer. The choice of treatment depended mainly on the stage of the disease. Treatments included surgery and chemotherapy (medicines to treat cancer).

The sponsor has provided sufficient information to show that human MHC non-restricted cytotoxic T-cell line might be of significant benefit for patients with ovarian cancer because early studies indicate that it might improve the treatment of patients with this condition. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Human MHC non-restricted cytotoxic T-cell line is an advanced therapy medicine that belongs to the group called 'somatic cell therapy products'. These are medicines that contain cells or tissues that have been manipulated so that they can be used to cure, diagnose or prevent a disease.
Human MHC non-restricted cytotoxic T-cell line is made up of a type of cells called cytotoxic T cells that are grown in the laboratory. Cytotoxic T cells form part of the immune system (the body's natural defences) and work by attacking diseased or infected cells in the body. In this medicine, the cells have been manipulated so that they are able to kill cancer cells. They also have the ability to discriminate between cancer cells and normal cells. This medicine is expected to kill the target cancer cells in different ways, including releasing cytotoxic (cell-killing) substances, activating the cell's processes that lead to it dying and stimulating the immune system to attack the cancer cells.

The effects of human MHC non-restricted cytotoxic T-cell line have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the product in patients with ovarian cancer were ongoing.

At the time of submission, human MHC non-restricted cytotoxic T-cell line was not authorised anywhere in the EU for ovarian cancer 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 7 October 2009 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.