Refusal of orphan designation for the treatment of cystic fibrosis


Table of contents


On 5 November 2009, the Committee for Orphan Medicinal Products (COMP) adopted a negative opinion on the orphan designation application for molgramostim for the treatment of cystic fibrosis. A negative decision was issued by the European Commission on 16 July 2010.

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 reason:

The sponsor had not provided sufficient evidence to justify the claim that molgramostim might be of significant benefit for patients with cystic fibrosis. The sponsor provided preclinical data from an animal model that was not relevant to the condition and anecdotal data from only one cystic fibrosis patient. These data were not considered sufficient to show that the product for which designation was applied might have a clinically relevant advantage over existing treatments or that the product might represent a major contribution to patient care. This justification was necessary because other satisfactory methods of treatment for cystic fibrosis had already been authorised in the European Union.

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 cystic fibrosis
Orphan designation status
EU designation number
Date of refusal of designation
Drugrecure Aps
c/o Serendex ApS
Cobis Building
Ole Maaløes vej 3
2200 Copenhagen
Telephone: +45 43 58 37 59
Telefax: +45 43 58 37 69

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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