EU/3/16/1791

Ovarian cancer is cancer of the ovaries, the 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 debilitating and life-threatening disease that is associated with poor long-term survival.

At the time of designation, ovarian cancer affected approximately 3.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 170,000 people¹, 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)

¹Disclaimer: 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 28), Norway, Iceland and Liechtenstein. This represents a population of 513,700,000 (Eurostat 2016).

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 results from early studies suggest that, when used in patients who have already had initial treatment, the medicine may improve the length of time patients live without their disease getting worse. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Ovarian cancer cells often have increased levels of a protein called survivin, which is involved in the regulation of cell death (apoptosis). In ovarian cancer cells, large amounts of survivin help prevent cell death; this results in the cancer cells surviving much longer than normal cells and multiplying in an uncontrolled fashion.

This medicine works by stimulating the patient's immune system, the body's natural defences, so that it targets and destroys the cancer cells. The medicine contains short fragments of survivin protein. When given to the patient, the patient's immune system is expected to recognise survivin as 'foreign'. This is expected to lead the immune system to attack and destroy ovarian cancer cells overproducing survivin.

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

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

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

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 October 2016 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.