Refusal of orphan designation for treatment of complex regional pain syndrome

zoledronic acid

Overview

On 6 February 2013, the Committee for Orphan Medicinal Products (COMP) adopted a negative opinion on the orphan designation application for zoledronic acid for the treatment of complex regional pain syndrome (CRPS). A negative decision was issued by the European Commission on 17 April 2013.

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 data submitted by the sponsor were not considered sufficient to demonstrate that the medicine could plausibly be used in the treatment of CRPS. The main data provided in support of the application was obtained from a conference abstract reporting on a non-sponsor-generated study that lacked relevant details that would be necessary for an in-depth assessment of the results.

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: Zoledronic acid for the treatment of complex regional pain syndrome

Adopted Reference Number: EMA/COMP/102965/2013

Key facts

• Active substance:
  zoledronic acid
• Intended use:
  Treatment of complex regional pain syndrome
• Orphan designation status:
  Negative
• EU designation number:
  -
• Date of refusal of designation:
  17/04/2013
• Sponsor:

  Axsome Therapeutics Limited; 88 Wood Street; London EC2V 7RS; United Kingdom; Tel. [phone]; Fax [phone]; 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.