EU/3/11/900: 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 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 152,000 people, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the 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 506,300,000 (Eurostat 2011).

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 how advanced the disease was. Treatments included surgery and chemotherapy (medicines to treat cancer).

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with ovarian cancer because it is more difficult to break down by the body which prolongs its action in tumour tissues, as supported by early studies, and might improve the outcome of patients. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

This medicine is a pro-drug that contains the active substance irinotecan, which has been 'pegylated' (attached to a chemical called polyethylene glycol). When it is given to a patient, the medicine is broken down by the body into irinotecan and a chemical called 7-ethyl-10 hydroxycampothecin (SN38). Irinotecan and SN38 block the action of an enzyme, topoisomerase I, which is involved in the division of cell DNA. When the enzyme is blocked, the DNA strands break, and this prevents the cancer cells from dividing and they eventually die. The fact that the medicine is pegylated is expected to decrease the rate at which the irinotecan is removed from the body, allowing it to act for longer.

The effects of this medicine have been evaluated in experimental models.

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

At the time of submission, this medicine was not authorised anywhere in the EU for ovarian cancer. Orphan designation of this medicine had been granted in the United States for the treatment of ovarian cancer.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 July 2011 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 EU) 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.