EU/3/03/155

Tumours that begin in the ovaries are known as ovarian tumours. Tumours that have potential to grow rapidly and infiltrate surrounding healthy tissues are called ovarian cancers. Due to the absence of symptoms in early stages of the disease, the majority of patients are diagnosed when the tumours have spread locally or to distant parts of the body. Ovarian cancer is a life-threatening condition.

At the time of designation, ovarian cancer affected approximately 2.4 in 10,000 persons in the European Union (EU). This was equivalent to a total of 92,000 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. At the time of designation, this represented a population of 382,800,000 (Eurostat 2003).

Several anticancer medicinal products had been authorised for the condition in the Community at the time of submission of the application for orphan designation. Although a significant percentage of patients respond to the initial chemotherapy (using drugs to kill cancer cells), most ovarian cancers grow again and respond moderately or poorly to subsequent chemotherapy. ACA125 might be of potential significant benefit for the treatment of ovarian cancer, because it may offer a new mechanism of action to fight the disease. This assumption will have to be confirmed. This will be necessary to maintain the orphan status.

Antibodies are proteins in the body that target specific shapes on the surface of foreign bodies, such as bacteria or cancer cells. CA125 is a protein that can be found on the surface of the ovarian cancer cells. ACA125 is a special type of antibody that mimicks CA125, and this is in turn expected to trigger the production of other antibodies against CA125. Antibodies such as ACA125 that trigger the production of other antibodies are called 'anti-idiotypic' antibodies. This anti-idiotypic antibody is shaped as a 'double mirror image' of the CA125 protein. By mimicking the CA125 protein, ACA125 may help the body's immune system to attack and kill the cancer cells.

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

At the time of submission, ACA125 was not marketed anywhere worldwide 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 13 June 2003 recommending the granting of this designation.

• the seriousness of the condition;
• the existence or not of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or insufficient return on development investments.

Designated orphan medicinal products are still-investigational products that are considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy will be necessary before this product can be granted a marketing authorisation.