EU/3/18/2116: Orphan designation for the treatment of cystic fibrosis

Ivacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide, tezacaftor

Overview

This medicine is now known as ivacaftor / tezacaftor / elexacaftor.

On 14 December 2018, orphan designation (EU/3/18/2116) was granted by the European Commission to Vertex Pharmaceuticals (Europe) Limited, United Kingdom, for ivacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide, tezacaftor for the treatment of cystic fibrosis.

The sponsorship was transferred to Vertex Pharmaceuticals (Ireland) Limited, Ireland, in February 2019.

Ivacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide, tezacaftor has been authorised in the EU as Kaftrio since 21 August 2020.

The sponsor’s address was updated in January 2022.

Key facts

Active substance
Ivacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide, tezacaftor
Intented use
Treatment of cystic fibrosis
Orphan designation status
Positive
EU designation number
EU/3/18/2116
Date of designation
14/12/2018
Sponsor

Vertex Pharmaceuticals (Ireland) Limited
Unit 49
Northwood Court
Block F2 Santry
Dublin 9
Ireland
E-mail: vertexmedicalinfo@vrtx.com

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Kaftrio at the time of marketing authorisation in 2020 and at the time of change to the terms of the marketing authorisation in 2021 (II-0001), and confirmed that the orphan designation should be maintained.

More information is available in the PDF icon orphan medicine assessment report and PDF icon orphan maintenance assessment report II-0001

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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