EU/3/04/193

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Orphan designation

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.

In January 2014, Fresenius Biotech GmbH changed name to Neovii Biotech GmbH.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

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 is the estimated number of patients affected by the condition?

At the time of designation, ovarian cancer affected approximately 2.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 111,0000 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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 464,200,000 (Eurostat 2004).

What treatments are 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.

How is this medicine expected to work?

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 medicine?

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.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 5 February 2004 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:
  • 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.

Key facts

Product details for <p>Anti-epithelial cell adhesion molecule/anti-CD3 monoclonal antibody</p>
Active substanceAnti-epithelial cell adhesion molecule/anti-CD3 monoclonal antibody
Medicine Name
Disease/conditionTreatment of ovarian cancer
Date of decision07/03/2004
OutcomePositive
Orphan decision numberEU/3/04/193

Review of designation

Sponsor’s contact details:

Neovii Biotech GmbH
Am Haag 6-7
D-82166 Graefelfing
Germany
Tel. +49 89 89 88 88 260
Fax +49 89 89 88 88 17
E-mail: regulatory@fresenius-biotech.com

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.