EU/3/08/556

About

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.

Key facts

Active substance
N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide (ivacaftor)
Medicine name
Kalydeco
Disease / condition
Treatment of cystic fibrosis
Date of first decision
08/07/2008
Outcome
Positive
EU designation number
EU/3/08/556

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, and confirmed that the orphan designation should be maintained.

More information is available in the PDF iconorphan maintenance assessment report

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.

Sponsor's contact details

Vertex Pharmaceuticals (Ireland) Limited
28-32 Pembroke Street Upper
Dublin 2
D02 EK84
Ireland
Tel. +353 1761 7296
E-mail: vertexmedicalinfo@vrtx.com

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;

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