EU/3/12/1049: 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 not more than 2.1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of not more than 106,000 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, particularly platinum-based medicines).

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for some patients with ovarian cancer, based on early data showing clinical response in ovarian cancer patients who have a mutation in a gene called 'BRCA' and those whose cancers are resistant to platinum-based treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Rucaparib is expected to work by blocking the activity of a family of proteins called poly(ADP-ribose) polymerases (PARPs). These proteins play an important role in the repair of DNA in cells. Some cancer cells, including those with a mutation in the BRCA gene, are particularly dependent on PARPs to repair damaged DNA because they lack other proteins for repairing DNA that normal cells have.

By blocking the activity of PARPs in cancer cells, rucaparib is expected to stop the cancer cells from being able to repair damaged DNA and this eventually leads to the death of the cancer cells, thereby slowing down the growth of the cancer.

The effects of rucaparib have been evaluated in experimental models.

At the time of submission of the application for orphan designation, preliminary clinical trials with rucaparib including patients with ovarian cancer were ongoing.

At the time of submission, rucaparib was not authorised anywhere in the EU for ovarian cancer. Orphan designation of rucaparib has been granted in the United States of America for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 5 September 2012 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.