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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.

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 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 was equivalent to a total of around 145,000 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 on the basis of data from the European Union (EU 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 500,300,000 (Eurostat 2007).

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 07/10/2014

Key facts

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

Review of designation

Related information

Sponsor’s contact details

AstraZeneca AB
151 85  Södertälje
Tel. +44 (0)1625 514776 (AstraZeneca UK Limited)

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.