Refusal of orphan designation for the treatment of uraeumic pruritus

Nalbuphine hydrochloride


On 27 February 2015, the Committee for Orphan Medicinal Products (COMP) adopted a negative opinion on the orphan designation application for nalbuphine hydrochloride for the treatment of uraemic pruritus (itching associated with chronic kidney disease). A negative decision was issued by the European Commission on 3 July 2015.

The sponsor applied for orphan designation on the basis of the seriousness and the rarity of the condition.

The negative opinion was based on the following reason:

  • the sponsor failed to establish that uraemic pruritus is a distinct, recognisable medical entity with signs and symptoms that can be distinguished from pruritus caused by other conditions.

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, including its use in clinical trials. A marketing authorisation can still be obtained if quality, safety and efficacy are demonstrated.

Key facts

Active substance
Nalbuphine hydrochloride
Intended use
Treatment of uraemic pruritus
Orphan designation status
EU designation number
Date of refusal of designation
Trevi Therapeutics Limited
Morton House, 9 Beacon Court
Pitstone Green Business Park
Pitstone LU7 9GY
United Kingdom
Tel. + 44 (0)7775 824952
Fax + 44 (0)1296 661213

Review of designation

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

Documents related to this orphan designation evaluation

Patients' organisations

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

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

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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