On 8 July 2008, orphan designation (EU/3/08/556) was granted by the European Commission to Voisin Consulting S.A.R.L., France, for N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide for the treatment of cystic fibrosis.
The sponsorship was transferred to Vertex Pharmaceuticals (U.K.) Limited, United Kingdom, in August 2011 and to Vertex Pharmaceuticals (Europe) Limited, United Kingdom, in August 2015.
N-(2,4-Di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide has been authorised in the EU as Kalydeco since 23 July 2012.
This medicine is now known as ivacaftor.
The sponsorship was transferred to Vertex Pharmaceuticals (Ireland) Limited, Ireland, in November 2018.
The sponsor’s address was updated in January 2022.
Treatment of cystic fibrosis
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Kalydeco at the time of change to the terms of the marketing authorisation in 2018 (II-0063), in 2020 (II-0085) and in 2021 (II-0089), and confirmed that the orphan designation should be maintained.
During its meeting of 12 and 13 June 2012, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/08/556 for Kalydeco (ivacaftor)1 as an orphan medicinal product for the treatment of cystic fibrosis. 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 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. The COMP recommended that the orphan designation of the medicine be maintained2.
1At time of orphan designation, ivacaftor was known as N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide.
2The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 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 cannot be placed on the market.
Documents related to this orphan designation evaluation
EU/3/08/556: Public summary of positive opinion for orphan designation of N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide for the treatment of cystic fibrosis (PDF/207.66 KB)
First published: 02/04/2009
Last updated: 10/08/2022
Recommendation for maintenance of orphan designation at the time of marketing authorisation: Kalydeco (ivacaftor) for the treatment of cystic fibrosis (PDF/118.72 KB)
First published: 31/08/2012
Last updated: 10/08/2022
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: