Refusal of orphan designation for the treatment of complex regional pain syndrom

zoledronic acid


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.

Key facts

Active substance
zoledronic acid
Intended use
Treatment of complex regional pain syndrome
Orphan designation status
EU designation number
Date of refusal of designation
Axsome Therapeutics Limited
88 Wood Street
London EC2V 7RS
United Kingdom
Tel. +44 (0)2036 171582
Fax +44 (0)2080 432268

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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