Kalydeco - opinion on variation to marketing authorisation

Opinion

EMA has issued an opinion on a change to this medicine's authorisation.

ivacaftor

Opinion

On 27 February 2025, 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 adopted extensions to the existing indications for Kalydeco film-coated tablets and granules in sachets to include treatment of patients with at least one non-class I CFTR mutation, as follows:¹

Kalydeco tablets are indicated:

• In a combination regimen with ivacaftor/tezacaftor/elexacaftor tablets for the treatment of adults, adolescents and children aged 6 years and older with cystic fibrosis (CF) who have at least one non-class I F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (see sections 4.2 and 5.1).

Kalydeco granules are indicated:

• In a combination regimen with ivacaftor/tezacaftor/elexacaftor for the treatment of cystic fibrosis (CF) in paediatric patients aged 2 to less than 6 years who have at least one non‑class I F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (see sections4.2 and 5.1).

For information, the full indications for Kalydeco will be as follows:

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 R117HCFTR 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/ivacaftor tablets for the treatment of adults, adolescents, and children aged 6 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+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272‑26A→G, and 3849+10kbC→T.
• In a combination regimen with ivacaftor/tezacaftor/elexacaftor tablets for the treatment adults, adolescents and children aged 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Kalydeco granules are indicated:

• As monotherapy for the treatment of infants aged at least 1 month, toddlers and children weighing 3 kg to less than 25 kg 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 ivacaftor/tezacaftor/elexacaftor for the treatment of cystic fibrosis (CF) in paediatric patients aged 2 to less than 6 years who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (see sections 4.2 and 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 on the EMA website in all official European Union languages after a decision on this change to the marketing authorisation has been granted by the European Commission.

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¹New text in bold, removed text as strikethrough

CHMP post-authorisation summary of positive opinion for Kalydeco WS-2551

AdoptedReference Number: EMA/CHMP/64893/2025

Key facts

• Name of medicine:
  Kalydeco
• EMA product number:
  EMEA/H/C/002494
• Active substance:
  ivacaftor
• International non-proprietary name (INN) or common name:
  ivacaftor
• Therapeutic area (MeSH):
  Cystic Fibrosis
• Anatomical therapeutical chemical (ATC) code:
  R07AX02
• 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.

• Marketing authorisation holder:
  Vertex Pharmaceuticals (Ireland) Limited
• Date of opinion:
  27/02/2025
• Status:
  Positive