Alyftrek

Opinion

EMA has issued an opinion on this medicine

deutivacaftor / tezacaftor / vanzacaftor

Overview

On 25 April 2025, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Alyftrek¹, intended for the treatment of cystic fibrosis in people aged 6 years and older who have at least one non‑class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The applicant for this medicinal product is Vertex Pharmaceuticals (Ireland) Limited.

Alyftrek will be available as 50 mg / 20 mg / 4 mg and 125 mg / 50 mg / 10 mg film-coated tablets. The active substances of Alyftrek are deutivacaftor / tezacaftor / vanzacaftor (ATC code: R07AX33). Vanzacaftor and tezacaftor, are CFTR correctors that bind to different sites on the CFTR protein, leading to an increase in the amount of CFTR protein on the cell surface; deutivacaftor improves the activity of the defective CFTR protein at the cell surface. These combined actions make lung mucus and digestive juices less thick, thereby helping to relieve symptoms of the disease.

The benefits of Alyftrek are improved lung function, as measured by percent predicted FEV1  (forced expiratory volume in one second), after 24 weeks of treatment. Two phase 3, randomised, double-blind clinical studies showed Alyftrek to be as effective as Kaftrio in patients with cystic fibrosis and non-class I mutations aged 12 years and older.

A supportive, single arm, open-label study in children aged 6-11 years was also submitted. Most children harboured an F508del mutation.

The most common side effects are headache and diarrhoea.

The full indication is:

Alyftrek tablets are indicated for the treatment of cystic fibrosis (CF) in people aged 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Alyftrek should be prescribed by physicians experienced in the treatment of cystic fibrosis.

Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published on the EMA website in all official European Union languages after the marketing authorisation has been granted by the European Commission.

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¹This product was designated as an orphan medicine during its development. EMA will now review the information available to date to determine if the orphan designation can be maintained

CHMP summary of positive opinion for Alyftrek

AdoptedReference Number: EMA/CHMP/131190/2025

Product details

• Name of medicine:
  Alyftrek
• Active substance:
  • deutivacaftor
  • tezacaftor
  • vanzacaftor calcium dihydrate
• International non-proprietary name (INN) or common name:
  deutivacaftor / tezacaftor / vanzacaftor
• Therapeutic area (MeSH):
  Cystic Fibrosis
• Anatomical therapeutic chemical (ATC) code:
  R07AX33
• EMA product number:
  EMEA/H/C/006382
• 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.

• Marketing authorisation applicant:
  Vertex Pharmaceuticals (Ireland) Limited
• Opinion adopted:
  25/04/2025
• Opinion status:
  Positive