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.

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.

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.

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 12/03/2015

Key facts

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

Review of designation

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*.


*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.

Life-threatening or long-term debilitating nature of the condition

The Committee for Medicinal Products for Human Use (CHMP) recommended the authorisation of Lynparza for:

‘maintenance treatment, as monotherapy, 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’.

This falls within the scope of the product’s designated orphan indication, which is: ‘ovarian cancer’.

The COMP concluded that there had been no change in the seriousness of the condition since the orphan designation in 2012. Ovarian cancer remains a long-term debilitating and life-threatening condition that is associated with shortened life expectancy.

Prevalence of the condition

The sponsor provided updated information on the prevalence of ovarian cancer based on data from the database GLOBOCAN (2012) and data from the scientific literature.

On the basis of the information provided by the sponsor and the knowledge of the COMP, the COMP concluded that the prevalence of ovarian cancer remains below the ceiling for orphan designation, which is 5 people in 10,000. At the time of the review of the orphan designation, the prevalence was estimated to be approximately 3 people in 10,000. This is equivalent to a total of around 153,000 people in the EU.

Existence of other methods of treatment

The COMP noted that, at the time of the review of the orphan designation, the main treatments for ovarian cancer were surgery and/or medicines to treat cancer, which might include platinum–based therapy.

Significant benefit of Lynparza

The COMP concluded that the claim of a significant benefit of Lynparza in ovarian cancer is justified because, in patients with a BRCA mutation whose cancer has come back following treatment with platinum–based medicines, Lynparza can be given to maintain the response to additional treatment with platinum–based medicines.

This is based on a main study which showed that, for patients with a BRCA mutation whose cancer has come back after treatment, the time patients lived without the disease getting worse after further platinum treatment was 11.2 months with Lynparza compared with 4.3 months in patients given placebo.

Therefore, although other methods for the treatment of this condition have been authorised in the EU, the COMP concluded that Lynparza is of significant benefit for patients affected by ovarian cancer.

Conclusions

Based on the data submitted and the scientific discussion within the COMP, the COMP considered that olaparib still meets the criteria for designation as an orphan medicinal product and that Lynparza should remain in the Community Register of Orphan Medicinal Products.

Further information on the current regulatory status of Lynparza can be found in the European public assessment report (EPAR).

Related information

Sponsor’s contact details

AstraZeneca AB
151 85  Södertälje
Sweden
Tel. +44 (0)1625 514776 (AstraZeneca UK Limited)
E-mail: olaparib@astrazeneca.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.