EU/3/07/478

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Orphan designation

On 14 September 2007, orphan designation (EU/3/07/478) was granted by the European Commission to Pharmion Ltd, UK, for N-(2-amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide for the treatment of Hodgkin's lymphoma.

The sponsorship was transferred to Celgene Europe Limited, United Kingdom, in November 2008 and subsequently to CanReg (Europe) Limited, Ireland, in March 2009.

What is Hodgkin's lymphoma?

Hodgkin’s lymphoma is a type of cancer that develops in the lymphatic system. The lymphatic system is part of the body’s immune system: the body's natural defence against infection and disease. It is a complex system made up of organs such as bone marrow (the spongy tissue inside the large bones in the body), thymus and spleen, and a network of lymph nodes throughout the body that are connected by lymphatic vessels. As lymphatic tissue is found throughout the body, Hodgkin's lymphoma can begin in almost any part of the body and can spread to almost any tissue or organ in the body. Normally, the growth and duplication of lymphatic cells takes place in a controlled manner, however in Hodgkin’s lymphoma this process is out of control and the cells continue to divide, developing into a tumour. Hodgkin's lymphoma can occur in both adults and children; however, treatment for adults may be different from treatment for children. Hodgkin's lymphoma can usually be cured, if found and treated early. Despite the available treatments Hodgkin’s lymphoma remains a serious and life-threatening condition in certain patients.

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

At the time of designation, Hodgkin's lymphoma affected approximately 2.4 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 120,000 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 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. This represents a population of 498,000,000 (Eurostat 2006).

What treatments are available?

Three different types of standard treatment are available and used: chemotherapy (using drugs to kill cancer cells), radiotherapy (using high-energy x-rays or other types of high-energy rays to kill cancer cells) and surgery (removing all possible cancer tissue in an operation). Bone marrow transplantation is also used. Several treatments had been authorised at the time of submission of the application for orphan drug designation.

N-(2-amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide could be of potential significant benefit for the treatment of Hodgkin’s lymphoma, because it may act in a different way to other types of treatment. Further, the product can be given orally three times per week, instead of as an injection (as is necessary for many of the currently authorised products). This assumption of significant benefit will have to be confirmed at the time of marketing authorisation, as this is necessary to maintain the orphan status.

How is this medicine expected to work?

N-(2-amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide acts by blocking the action of a group of enzymes (a protein that speeds biochemical reactions in the body) called histone deacetylases, which are involved in the proliferation of cancer cells. By blocking some of the subtypes of this enzyme, the product is expected to block cancer growth.

What is the stage of development of this medicine?

The effects of N-(2-amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with Hodgin’s lymphoma were ongoing.

N-(2-amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide was not authorised anywhere in the world for treatment of Hodgkin's lymphoma, or designated as orphan medicinal product anywhere 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 25 July 2007 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;
  • either the rarity of the condition (affecting not more than 5 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>N-(2-amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide</p>
Active substanceN-(2-amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide
Medicine Name
Disease/conditionTreatment of Hodgkin's lymphoma
Date of decision14/09/2007
OutcomePositive
Orphan decision numberEU/3/07/478

Review of designation

Sponsor’s contact details:

CanReg (Europe) Limited
Templetown
Carlingford
County Louth
Ireland
Telephone: +353 42 937 67 40
Telefax: +353 42 937 67 40

Patients’ associations contact points:

Leukaemia Care
2 Shrubbery Avenue
Worcester
WR1 1QH
United Kingdom
0800 169 6680 (freephone for UK residents)
Telephone: +44 19 05 33 00 03 / + 44 84 57 67 32 03
Telefax: +44 19 05 33 00 90
E-mail: enquiries@leukaemiaCARE.org.uk

Ligue Nationale Contre le Cancer
13 Av. de la Grande Armee
75116 Paris
France
Telephone: +33 1 45 00 00 17
Tefefax: +33 1 45 00 63 06
E-mail: ligue@ligue-cancer.net

Associazione Italiana contro le Leucemie-linfomi e mieloma ONLUS
Via Ravenna 34
00161 Roma
Italy
Telephone: +39 06 44 03 763
Telefax: +39 06 44 04 226
E-mail: ail@ail.it