EU/3/08/535

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Orphan designation

On 1 April 2008 , orphan designation (EU/3/08/535) was granted by the European Commission to Chiltern International Limited, United Kingdom, for humanised monoclonal antibody to the folate receptor alpha for the treatment of ovarian cancer.

The sponsorship was transferred to Eisai Europe Limited, United Kingdom, in June 2010.

What is ovarian cancer?

Tumours that begin in the ovaries are known as ovarian tumours. Tumours which have the 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 2.9 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 144,000 people, and is below the threshold 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 based on data from the European Union (EU 27), Norway, Iceland and Lichtenstein. This represents a population of 498,000,000 (Eurostat 2006). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

What treatments are available?

Several anticancer medicinal products were authorised for the condition in the Community at the time of submission of the application for orphan designation. Although a significant proportion of patients respond to the initial chemotherapy (drugs used to kill cancer cells), most ovarian cancers grow again and respond moderately or poorly to subsequent chemotherapy.

Humanised monoclonal antibody to the folate receptor alpha might be of potential significant benefit for the treatment of ovarian cancer. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

A large majority of ovarian tumour cells bear a molecule called the folate receptor alpha (a protein that binds folic acid) on the surface of the cell. Humanised monoclonal antibody to the folate receptor alpha attracts normal human blood monocytes, a type of white blood cells that are a part of the immune system. Monocytes are capable of ingesting foreign substances and killing infected cells. The antibody is expected to attract monocytes that may then attack and kill folate receptor alpha-bearing tumour cells, such as cancer cells. This is expected to reduce or to stop the tumour growth.

What is the stage of development of this medicine?

The effects of humanised monoclonal antibody to the folate receptor alpha were evaluated in experimental models.

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

Humanised monoclonal antibody to the folate receptor alpha was not authorised anywhere worldwide for treatment of ovarian cancer, at the time of submission. 

Orphan designation of humanised monoclonal antibody to the folate receptor alpha was granted in the United States for treatment of ovarian cancer.

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

Opinions on orphan medicinal products designations are based on the following cumulative criteria:
  • the seriousness of the condition, 
  • the existence of alternative methods of diagnosis, prevention or treatment and
  • either the rarity of the condition (affecting not more than 5 in 10,000 people in the European Union) 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>Humanised monoclonal antibody to the folate receptor alpha</p>
Active substanceHumanised monoclonal antibody to the folate receptor alpha
Medicine Name
Disease/conditionTreatment of ovarian cancer
Date of decision01/04/2008
OutcomePositive
Orphan decision numberEU/3/08/535

Review of designation

Sponsor’s contact details:

Eisai Europe Limited
European Knowledge Centre 
Mosquito Way 
Hatfield, Herts AL10 9SN 
United Kingdom
Telephone: +44 (0) 845 676 5089
Telefax: +44 (0) 845 676 1401

Patients’ associations contact points:

Cancer BACUP
3 Bath Place
Rivington Street
London
EC2A 3JR
United Kingdom
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 (LNCC)
12, rue Corvisart
75013 Paris
France
Telephone : +33 1 53 55 24 00
Telefax : +33 1 43 36 91 10
E-mail: ligue@ligue-cancer.net