EU/3/20/2266: Orphan designation for the treatment of ovarian cancer

Ovarian cancer is cancer of the ovaries, the two organs in the female reproductive system that produce eggs. Most ovarian cancers occur in women aged over 50 years. Due to the absence of clear symptoms in the early stages of the disease, it is usually 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 4.9 in 10,000 people in the European Union (EU). This was equivalent to a total of around 254,000 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).

* 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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).

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 the medicine might be of significant benefit for patients with ovarian cancer because early results suggest that the medicine used together with authorised treatments provides greater benefits to patients whose cancer had come back or as initial treatment before surgery than the authorised treatments alone.

This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

The medicine contains 2 genes responsible for making a protein called interleukin-12 (IL-12) which stimulates the immune system (the body’s natural defences). When injected locally, next to the ovarian cancer, the genes are expected to produce IL-12 which will in turn make the immune system more active and encourage it to attack the cancer cells. This should help reduce the size of the cancer.

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 the treatment of ovarian cancer. Orphan designation of the medicine had been granted in the United States for the condition.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 19 March 2020, 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.