Refusal of orphan designation for the treatment of amyotropic lateral sclerosis


Table of contents


On 16 January 2011, the Committee for Orphan Medicinal Products (COMP) adopted a negative opinion on the orphan designation application for nabilone for the treatment of amyotrophic lateral sclerosis. A negative decision was issued by the European Commission on 16 November 2011.

The sponsor applied for orphan designation on the basis of the seriousness and the rarity of the condition, as well as an assumption of potential benefit over currently available methods of treatment.

The negative opinion was based on the following reasons:

  • The sponsor had not provided data on the effect of nabilone in amyotrophic lateral sclerosis.
  • Without data on the effect of nabilone in amyotrophic lateral sclerosis, a potential significant benefit of nabilone over currently available methods of treatment could not be established.

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
Intended use
Treatment of amyotrophic lateral sclerosis
Orphan designation status
EU designation number
Date of refusal of designation
MRN-Medical Research Network GmbH
Postgasse 11/22
1010 Vienna
Telephone: +43 676 566 78 04
Telefax: +43-01 470 70 50 15

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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