EU/3/08/590

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Orphan designation

On 5 December 2008, orphan designation (EU/3/08/590) was granted by the European Commission to AstraZeneca AB, Sweden, for 2-[[3-({4-[(5-{2-[(3-fluorophenyl)amino]-2-oxoethyl}-1H-pyrazol-3-yl)amino]-quinazolin-7-yl}oxy)propyl](ethyl)amino]ethyl dihydrogen phosphate trihydrate for the treatment of acute myeloid leukaemia.

What is acute myeloid leukaemia?

Acute myeloid leukaemia (AML) is a cancer of the white blood cells. In this disease, the bone marrow produces large numbers of abnormal, immature white blood cells called ‘blasts’. These abnormal cells quickly build up in large numbers in the bone marrow and are found in the blood.

AML is life-threatening because these immature cells take the place of the normal white blood cells. As a result, the patient’s ability to fight diseases is reduced.

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

At the time of designation AML affected less than 2 in 10,000 people in the European Union (EU) *. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 100,000 people.

*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 Liechtenstein. This represents a population of 502,282,000 (Eurostat 2008).

What treatments are available?

Treatment for AML is complex and depends on a number of factors including the extent of the disease, whether it has been treated before, and the patient’s age, symptoms and general state of health. The primary treatment for AML is chemotherapy (using medicines to kill cancer cells).

The sponsor has provided sufficient information to show that 2-[[3-({4-[(5-{2-[(3-fluorophenyl)amino]-2-oxoethyl}-1H-pyrazol-3-yl)amino]-quinazolin-7-yl}oxy)propyl](ethyl) amino]ethyl dihydrogen phosphate trihydrate might be of significant benefit for the treatment of AML because it may be more effective than other medicines for this disease. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine product expected to work?

2-[[3-({4-[(5-{2-[(3-fluorophenyl)amino]-2-oxoethyl}-1H-pyrazol-3-yl)amino]-quinazolin-7-yl}oxy) propyl](ethyl)amino]ethyl dihydrogen phosphate trihydrate is expected to work by blocking enzymes called ‘aurora kinases’. These enzymes play an essential role in controlling the movement of the chromosome separation when the cells divide. By disrupting cell division, this medicine is expected to reduce the division of cancerous cells in AML.

What is the stage of development of this medicine?

The effects of 2-[[3-({4-[(5-{2-[(3-fluorophenyl)amino]-2-oxoethyl}-1H-pyrazol-3-yl)amino]-quinazolin-7-yl}oxy)propyl](ethyl)amino]ethyl dihydrogen phosphate trihydrate have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with acute myeloid leukaemia were ongoing.

At the time of submission, 2-[[3-({4-[(5-{2-[(3-fluorophenyl)amino]-2-oxoethyl}-1H-pyrazol-3-yl)amino]-quinazolin-7-yl}oxy)propyl](ethyl)amino]ethyl dihydrogen phosphate trihydrate was not authorised anywhere in the world for AML or designated as orphan medicinal product for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 October 2008 recommending the granting of this orphan 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 five in 10,000 people in the Community) 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>2-[[3-({4-[(5-{2-[(3-Fluorophenyl)amino]-2-oxoethyl}-1H-pyrazol-3-yl)amino]-quinazolin-7-yl}oxy)propyl](ethyl)amino]ethyl dihydrogen phosphate trihydrate</p>
Active substance2-[[3-({4-[(5-{2-[(3-Fluorophenyl)amino]-2-oxoethyl}-1H-pyrazol-3-yl)amino]-quinazolin-7-yl}oxy)propyl](ethyl)amino]ethyl dihydrogen phosphate trihydrate
Medicine Name
Disease/conditionTreatment of acute myeloid leukaemia
Date of decision04/12/2008
OutcomePositive
Orphan decision numberEU/3/08/590

Review of designation

Sponsor’s contact details:

AstraZeneca AB
Forskargatan 18
Sodertalje
SE-15185 Sweden
Telephone: + 46 8 553 275 91
Telefax: + 46 8 553 269 04
E-mail: orphandrugs@astrazeneca.com

Patients’ associations contact points:

Leukaemia CARE
One Birch Court
Blackpole East
Worcester
Worcestershire WR3 8SG
United Kingdom
Telephone: +44 1905 755977 / +44 845 521 3456
24 hour CARE line 0800 169 6680 (UK residents only)
Telefax: +44 1905 755166
E-mail: info@leukaemiacare.org.uk

Ligue Nationale Contre le Cancer
14 Rue Corvisart
75013 Paris
France
Telephone: +33 1 53 55 24 00
Telefax: +33 1 43 36 91 10
E-mail: ligue@ligue-cancer.net

Deutsche Krebshilfe e. V.
Buschstr. 32
53113 Bonn
Germany
Telephone: +49 2 287 29 900
Telefax: +49 2 287 29 90 11
E-mail: deutsche@krebshilfe.de