Kalydeco

ivacaftor

Table of contents

Opinion

On 17 September 2020, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the medicinal product Kalydeco. The marketing authorisation holder for this medicinal product is Vertex Pharmaceuticals (Ireland) Limited.

The CHMP recommended the approval of a new 75-mg strength for Kalydeco tablets and an extension to the existing indication in combination with Symkevi (tezacaftor/ivacaftor) to allow use in children from 6 years of age.

For information, the full indication for Kalydeco tablets will be as follows:1

Kalydeco tablets are indicated:

  • As monotherapy for the treatment of adults, adolescents, and children aged 6 years and older and weighing 25 kg or more with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R (see sections 4.4 and 5.1).
  • In a combination regimen with tezacaftor 100 mg/ivacaftor 150 mg tablets for the treatment of adults, and adolescents, and children aged 6 12 years and older with cystic fibrosis (CF) who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3AG, S945L, S977F, R1070W, D1152H, 2789+5GA, 3272 26AG, and 3849+10kbCT.
  • In a combination regimen with ivacaftor 75 mg/tezacaftor 50 mg/elexacaftor 100 mg tablets for the treatment of adults and adolescents aged 12 years and older with cystic fibrosis (CF) who are homozygous for the F508del mutation in the CFTR gene or heterozygous for F508del in the CFTR gene with a minimal function (MF) mutation (see section 5.1).

Detailed recommendations for the use of this product will be described in the updated summary of product characteristics (SmPC), which will be published in the revised European public assessment report (EPAR), and will be available in all official European Union languages after a decision on this change to the marketing authorisation has been granted by the European Commission.


1 New text in bold, removed text as strikethrough

Key facts

Name
Kalydeco
Agency product number
EMEA/H/C/002494
International non-proprietary name (INN) or common name
  • ivacaftor
Active substance
  • ivacaftor
Therapeutic area
Cystic Fibrosis
Accelerated assessment

This medicine had an accelerated assessment. This means that it is a medicine of major interest for public health, so its timeframe for review was 150 evaluation days rather than 210. For more information, see Accelerated assessment.

Orphan

This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Date opinion adopted
17/09/2020
Company name
Vertex Pharmaceuticals (Ireland) Limited
Status
Positive
Application type
Post-authorisation

How useful was this page?

Add your rating