Refusal of orphan designation for the prevention of graft rejection after liver transplantation


Table of contents


On 10 September 2008, the Committee for Orphan Medicinal Products (COMP) adopted a negative opinion on orphan designation of 4-[[[4-(4-Chlorophenoxy)phenyl]sulfonyl]-methyl]tetrahydro-N-hydroxy-2H-pyran-4- carboxamide for the prevention of graft rejection after liver transplantation. A negative decision was granted by the European Commission on 2 July 2009.

The sponsor applied for orphan designation on the basis of the seriousness and the rarity of the condition, as well as an assumption of potential benefit over currently available methods of treatment.

The negative opinion is based on the following reasons(s):

  • although satisfactory methods of prevention of the condition have been authorised in the Community, sufficient justification has not been provided that 4-[[[4-(4-Chlorophenoxy)phenyl]sulfonyl]-methyl]tetrahydro-N-hydroxy-2H-pyran-4-carboxamide may be of significant benefit to the population at risk of developing the condition.

Requests for designation as an orphan medicinal product are made for investigational products. Absence of orphan designation does not preclude the development of this product, in particular through clinical trials. A marketing authorisation can still be obtained if quality, safety and efficacy are demonstrated.

Key facts

Active substance
Intended use
Prevention of graft rejection after liver transplantation
Orphan designation status
EU designation number
Date of refusal of designation
Anthony William Fox
45 Cranham Gardens
Cranham, Essex
RM14 1JQ
United Kingdom
Telephone: +44 79 812 9553
Telefax: + 44 79 812 9553

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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