EU/3/06/355

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Orphan designation

On 17 February 2006, orphan designation (EU/3/06/355) was granted by the European Commission to Uppsala Medical Information System AB, Sweden, for zosuquidar trihydrochloride for the treatment of acute myeloid leukaemia. The sponsorship was transferred to Kanisa Europe Limited, United Kingdom, in May 2006.

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 acute myeloid leukaemia affected approximately 0.7 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 32,000 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 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

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).

How is this medicine expected to work?

Cancer cells develop several mechanisms of resistance to evade the toxic effects of anticancer drugs. One of these mechanisms involves a protein called multidrug resistance (MDR) transporter. MDR pumps substances out of the cell. In fact, MDR can pump anticancer agents out of the cancer cells. Therefore, the drugs cannot reach their target within the cancer cells and do not kill them. Zosuquidar does not have anticancer properties per se. However, it is an inhibitor of the MDR transporter and by blocking this transporter it is expected to help anticancer drugs to overcome this resistance mechanism and to exert their activity.

What is the stage of development of this medicine?

The effects of zosuquidar were 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.
Zosuquidar thihydrochloride was not authorised anywhere worldwide for acute myeloid leukaemia or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 11 January 2006 a positive opinion recommending the grant of the above-mentioned 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;
  • and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

Key facts

Product details for <p>Zosuquidar trihydrochloride</p>
Active substanceZosuquidar trihydrochloride
Medicine Name
Disease/conditionTreatment of acute myeloid leukaemia
Date of decision16/02/2006
OutcomePositive
Orphan decision numberEU/3/06/355

Review of designation

Sponsor’s contact details:

Kanisa Europe Limited
Mofo Notices Limited
C/Morrison & Foerster MNP
City Point, One Ropemaker Street
London, EC2Y 9AW
United Kingdom
Telephone: +44 207 920 4081
Telefax: +44 207 496 8581
E-mail: MODonnell@mofo.com

Patients’ associations contact points:

Leukaemia CARE
One Birch Court
Blackpole East
Worcester
Worcestershire WR3 8SG
United Kingdom
Telephone: +44 1905 75 59 77 / +44 845 521 34 56
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