EU/3/07/501

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Orphan designation

On 6 December 2007, orphan designation (EU/3/07/501) was granted by the European Commission to AstraZeneca AB, Sweden, for olaparib for the treatment of ovarian cancer.

What is ovarian cancer?

Tumours that begin in the ovaries are known as ovarian tumours. Tumours that have the potential to grow rapidly and invade 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.

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 medicinal products were 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 (drugs used to kill cancer cells), most ovarian cancers grow again and respond moderately or poorly to subsequent chemotherapy.

Olaparib 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?

Olaparib blocks the action of a protein called PARP (polyadenosine 5’-diphosphoribose polymerase). This protein helps repair damaged DNA (the genetic material in the cells); if PARP is blocked by olaparib, the damaged DNA in the tumour cell cannot be repaired, and the cell dies as a result. This is expected to reduce or block tumour growth.

What is the stage of development of this medicine?

The effects of olaparib 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.

Olaparib was not authorised anywhere in the world for ovarian cancer, or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 10 October 2007 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.

Name Language First published Last updated
EU/3/07/501: Public summary of positive opinion for orphan designation of olaparib for the treatment of ovarian cancer (English only) 02/07/2008 23/06/2010

Key facts

Product details for <p>Olaparib</p>
Active substanceOlaparib
Medicine Name
Disease/conditionTreatment of ovarian cancer
Date of decision06/12/2007
OutcomePositive
Orphan decision numberEU/3/07/501

Review of designation

Sponsor’s contact details:

AstraZeneca AB
Forskargatan 18
Sodertalje
SE-15185
Sweden
Telephone: +44 1625 51 47 76 (AstraZeneca UK Limited)
Telefax: +44 1625 51 09 08
E-mail: Olaparib@astrazeneca.com

Patient associations' contact points:

Ovacome
Elizabeth Garrett Anderson Hospital
London
WC1E 6DH
United Kingdom
Telephone: +44 20 73 80 95 89
E-mail: ovacome@ovacome.org.uk

Ligue Nationale Contre le Cancer
13 Av. de la Grande Armee
75116 Paris
France
Telephone: +33 1 45 00 00 17
Tefefax: +33 1 45 00 63 06
E-mail: ligue@ligue-cancer.net

The Association of European Cancer Leagues (ECL)
c/o Belgian Federation against Cancer
Chaussée de Louvain, 479
B-1030 Brussels
Belgium
Telephone: +32 2 743 3705
Telefax: +32 2 734 9250
E-mail: chartmann@cancer.be