Please note that this product (marketed as Pheburane) was withdrawn from the Community Register of designated orphan medicinal products in July 2013 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.
On 9 February 2012, orphan designation (EU/3/12/950) was granted by the European Commission to Lucane Pharma SA, France, for sodium phenylbutyrate for the treatment of ornithine-transcarbamylase deficiency.
Treatment of ornithine-transcarbamylase deficiency
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Review of designation
Please note that this product (marketed as Pheburane) was withdrawn from the Community register of designated orphan medicinal products in July 2013 on request of the sponsor, at the time of the granting of a marketing authorisation.
During its meeting of 12-13 April 2013, the Committee for Orphan Medicinal Products (COMP) reviewed the designations EU/3/12/951, EU/3/12/950 and EU/3/12/949 for Pheburane (sodium phenylbutyrate) as an orphan medicinal product for the treatment of the following urea-cycle disorders: carbamoyl-phosphate-synthase-1 deficiency, ornithine-transcarbamylase deficiency and citrullinaemia type 1.
The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other satisfactory methods of treatment. As other satisfactory methods of treatment for patients with this condition are authorised in the European Union (EU), the COMP also looked at the significant benefit of the product over existing treatments. As one of the criteria for orphan designation is no longer met (i.e. the significant benefit), the COMP recommended that the orphan designation of the product should not be maintained1.
The sponsor requested a re-examination of the COMP opinion. After considering the grounds for this request, the COMP re-examined the initial opinion, and confirmed the removal of the orphan designation on 1 July 2013.
1The removal of the orphan designation at time of marketing authorisation means that the product cannot benefit from 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with a comparable therapeutic indication can be placed on the market.
Recommendation for removal of orphan designation at the time of marketing authorisation: Pheburane (sodium phenylbutyrate) for the treatment of urea-cycle disorders (carbamoyl-phosphate-synthase-1 deficiency, ornithine-t... (PDF/89.51 KB)
First published: 08/08/2013
Last updated: 08/08/2013
Sponsor's contact details
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.