EU/3/07/501 - orphan designation for treatment of ovarian cancer

Olaparib

Overview

Please note that this product was withdrawn from the Community register of designated orphan medicinal products in March 2018 upon request of the marketing authorisation holder at the time of the granting of a change to the terms of the marketing authorisation.

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.

Olaparib has been authorised in the EU as Lynparza since 16 December 2014.

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).

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* 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.

Update: olaparib (Lynparza) has been authorised in the EU since 16 December 2014. Lynparza is indicated as monotherapy for the maintenance treatment of adult patients with platinum-sensitive relapsed BRCA-mutated (germline and/or somatic) high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete response or partial response) to platinum-based chemotherapy.

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.

EU/3/07/501: Public summary of positive opinion for orphan designation of olaparib for the treatment of ovarian cancer

AdoptedReference Number: EMEA/COMP/510787/2007 Rev. 3

Key facts

• Active substance:
  Olaparib
• Medicine name:
  Lynparza
• Intended use:
  Treatment of ovarian cancer
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/07/501
• Date of designation:
  06/12/2007
• Sponsor:

  AstraZeneca AB;

Review of designation

In March 2018 the Committee for Orphan Medicinal Products reviewed again the orphan designation of Lynparza at the time of the granting of a change to the terms of the marketing authorisation.

The sponsor formally requested the withdrawal of the orphan designation prior to the Committee's final opinion. More information is available in the Lynparza : Orphan designation withdrawal assessment report (post-authorisation).

During its meeting of 11 to 13 November 2014, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/07/501 for Lynparza (olaparib)as an orphan medicinal product for the treatment of ovarian cancer. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness of the condition and the existence of other methods of treatment. As other methods of treatment are authorised in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with ovarian cancer. The COMP recommended that the orphan designation of the medicine be maintained*.

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*The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with a comparable therapeutic indication cannot be placed on the market.