Refusal of orphan designation for the treatment of complex regional pain syndrom
zoledronic acid
Table of contents
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.
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. +44 (0)2036 171582 Fax +44 (0)2080 432268 E-mail: info@axsome.com |
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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: