On 8 March 2004, orphan designation (EU/3/04/193) was granted by the European Commission to Fresenius Biotech GmbH, Germany, for anti-epithelial cell adhesion molecule / anti-CD3 monoclonal antibody for the treatment of ovarian cancer.
- What is ovarian cancer?
Tumours that begin in the ovaries are known as ovarian tumours. Tumours which 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 the patients are diagnosed when the tumours have spread locally or to distant parts of the body. Ovarian cancer is a life-threatening condition.
- What are the methods of treatment available?
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. Anti-epithelial cell adhesion molecule / anti-CD3 monoclonal antibody 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.
- What is the estimated number of patients affected by the condition*?
According to the information provided by the sponsor, ovarian cancer was considered to affect about 90,000 persons in the European Union.
*Disclaimer: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 385,000,000 (Eurostat 2002) and may differ from the true number of patients affected by the condition. This estimate is based on available information and calculations presented by the sponsor at the time of the application.
- How is this medicinal product expected to act?
Antibodies are proteins in the body that target specific shapes on the surface of foreign bodies, such as bacteria or cancer cells. Anti-epithelial cell adhesion molecule / anti-CD3 monoclonal antibody is an antibody that has three locations where it can bind to specific shapes of different types of cells. The cells that it will recognise and bind are 1) the ovarian tumour cell, 2) the T cells, a type of cells that are part of the human body defence system and 3) other specific cells of the human body needed to activate these T cells. By physically binding these different types of cells, anti-epithelial cell adhesion molecule / anti- CD3 monoclonal antibody may help the body's immune system to attack and kill the ovarian cancer cells.
- What is the stage of development of this medicinal product?
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, anti-epithelial cell adhesion molecule / anti-CD3 monoclonal antibody was not marketed anywhere worldwide for ovarian cancer or designated as orphan medicinal product elsewhere for this condition.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 5 February 2004 a positive opinion recommending the grant of the above-mentioned designation.
- Opinions on orphan medicinal products designations are based on the following cumulative criteria:
- the seriousness of the condition,
- the existence or not of alternative methods of diagnosis, prevention or treatment and
- either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.
Designated orphan medicinal products are still investigational products which were considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy will be necessary before this product
can be granted a marketing authorisation.
|Active substance||Anti-epithelial cell adhesion molecule/anti-CD3 monoclonal antibody|
|Disease/condition||Treatment of ovarian cancer|
|Date of decision||07/03/2004|
|Orphan decision number||EU/3/04/193|
Review of designation
Sponsor’s contact details:
Fresenius Biotech GmbH
Am Haag 6-7
Telephone: +49 89 89 88 88 260
Telefax: +49 89 89 88 88 17
Patients’ associations contact points:
The Association of European Cancer Leagues (ECL)
c/o Belgian Federation against Cancer
Chaussée de Louvain, 479
Telephone: +32 2 743 3705
Telefax: +32 2 734 9250
3 Bath Place
Telephone: +44 20 76 96 90 03 / 0808 800 1234 (freephone for UK)
Telefax: +44 20 76 96 90 02
Ligue Nationale Contre le Cancer
13 Av. de la Grande Armee
Telephone: +33 1 45 00 00 17
Tefefax: +33 1 45 00 63 06