Refusal of orphan designation for treatment of uraemic pruritus

nalbuphine hydrochloride

Overview

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.

Public summary of opinion on orphan designation: Nalbuphine hydrochloride for the treatment of uraemic pruritus

Adopted Reference Number: EMA/COMP/504453/2015

Key facts

• Active substance:
  nalbuphine hydrochloride
• Intended use:
  Treatment of uraemic pruritus
• Orphan designation status:
  Negative
• EU designation number:
  -
• Date of refusal of designation:
  27/02/2015
• Sponsor:

  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; E-mail: [e-mail]

Review of designation

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