EU/3/12/1001

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Orphan designation

On 6 June 2012, orphan designation (EU/3/12/1001) was granted by the European Commission to Abbott Laboratories, United Kingdom, for 1-(4-{4-amino-7-[1-(2-hydroxyethyl)-1H-pyrazol-4-yl] thieno [3,2-c]pyridin-3-yl}phenyl)-3-(3-fluorophenyl)urea for the treatment of ovarian cancer.

The sponsorship was transferred to AbbVie Ltd, United Kingdom, in January 2013.

What is ovarian cancer?

Ovarian cancer is cancer of the ovaries (two organs in the female reproductive system that produce eggs). Most ovarian cancers occur in women over the age of 50 years. Due to the absence of symptoms in the early stages of the disease, the majority of patients are diagnosed when the cancer has spread to other parts of the body.

Ovarian cancer is a life-threatening disease that is associated with poor long-term survival.

What is the estimated number of patients affected by the condition?

At the time of designation, ovarian cancer affected not more than 3 in 10,000 people in the European Union (EU). This was equivalent to a total of not more than 153,000 people*, and is below the ceiling 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 509,000,000 (Eurostat 2012).

What treatments are available?

At the time of designation, several medicines were authorised in the EU for the treatment of ovarian cancer. The choice of treatment depended mainly on how advanced the disease was. Treatments included surgery and chemotherapy (medicines to treat cancer).

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with ovarian cancer because it works in a different way to existing treatments, and early studies in experimental models show that it may be of benefit when used alone and in combination with the anticancer medicine paclitaxel. Its use in combination with paclitaxel may offer an alternative to patients with ‘platinum-resistant’ ovarian cancer for whom existing treatments do not work. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

This medicine belongs to a group called ‘Aurora-kinase inhibitors’. These compounds work by blocking enzymes known as Aurora kinases, which play an important role in the control of cell division. Aurora kinases can be found in high amounts in cancer cells where they no longer function normally. By blocking these enzymes, the medicine is expected to stop the cancer cells from dividing.

The medicine is also expected to block enzymes known as tyrosine kinases, which are involved in the growth and spread of the cancer cells and in the development of new blood vessels that supply the tumours.

What is the stage of development of this medicine?

The effects of this medicinal product have been evaluated in experimental models.

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

At the time of submission, this medicinal product was not authorised anywhere in the EU for ovarian cancer or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 12 April 2012 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:
  • the seriousness of the condition;
  • the existence of alternative methods of diagnosis, prevention or treatment;
  • either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) 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>1-(4-{4-Amino-7-[1-(2-hydroxyethyl)-1H-pyrazol-4-yl] thieno [3,2-c]pyridin-3-yl}phenyl)-3-(3-fluorophenyl)urea</p>
Active substance1-(4-{4-Amino-7-[1-(2-hydroxyethyl)-1H-pyrazol-4-yl] thieno [3,2-c]pyridin-3-yl}phenyl)-3-(3-fluorophenyl)urea
Medicine Name
Disease/conditionTreatment of ovarian cancer
Date of decision06/06/2012
OutcomePositive
Orphan decision numberEU/3/12/1001

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor’s contact details

AbbVie Ltd
Abbott House
Vanwall Business Park
Vanwall Road
Maidenhead
Berkshire
SL6 4XE
United Kingdom
Tel. +44 (0)1628 644501
Fax +44 (0)1628 672624
E-mail: orphandrugs.enquiries@abbvie.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.