We are pleased to inform you that the new innovative treatment Alyftrek® (vanzacaftor/tezacaftor/deutivacaftor) for Cystic Fibrosis has been approved by the European Medicines Agency (EMA) on 1 July 2025. The new treatment is suitable for patients with Cystic Fibrosis aged 6 years and older who carry at least one non-class I (non-class I) mutation in the CFTR gene.
Alyftrek is a new generation CFTR modulator administered once daily with a fatty meal, offering a simplified treatment regimen and greater patient compliance. The new therapy has demonstrated non-inferior efficacy to Kaftrio (Trikafta) in improving lung function (FEV1) and superior reduction in sweat chloride levels; a marker of restoration of CFTR protein function.
Which patients are eligible for Alyftrek®
The treatment is aimed at people with at least one mutation in Classes II to VI (Class II-VI), i.e. mutations that allow the production or partial function of the CFTR protein. These mutations affect the vast majority of patients, many of whom are already receiving similar treatments.
The approval of Alyftrek in Europe offers a new treatment option for thousands of patients with simpler administration (1 time/day) and the prospect of improved quality of life, facilitating patient compliance.
As Prof. Marcus Mall, Professor of Paediatric Pulmonology and Director of the Cystic Fibrosis Centre at Charité Universitätsmedizin Berlin, said:
“The care of Cystic Fibrosis has been transformed with the advent of the highly effective CFTR modulator therapies and I am very pleased that we now have a new treatment option for even better management of this multisystemic disease. Deivacaftor/tezacaftor/vanzacaftor (Alyftrek) has shown that it can bring about a greater reduction of sodium chloride in sweat compared to existing treatments. By bringing more people closer to normal levels of CFTR protein function, this new drug has the potential to further improve outcomes for patients.”
This statement highlights the potential of the new treatment to enhance the long-term management of the disease.
What applies to patients with two Class I mutations
Alyftrek is not indicated for patients who have two Class I mutations, which completely block the production of the CFTR protein. This is a small group of patients for whom research projects for future treatments are already underway.
🎯 At the Panhellenic Cystic Fibrosis Association we remain firmly committed to ensuring equal access to all available treatments for every person with Cystic Fibrosis in Greece.
We recognise how difficult the wait for appropriate treatment can be and we stand by every patient who is still waiting.
The march towards “Breathless Breath” continues, until the day when there is a treatment option available for every Cystic Fibrosis patient, regardless of mutation.
* The new drug was also presented at the European Cystic Fibrosis Congress (ECFS 2025) in Milan, where our Association actively participated. Details of the conference will follow in a next announcement soon.
⚠️ The Association does not provide medical advice and is not involved in the selection of treatment regimens. For any questions, patients can contact their treating physicians.
Source: Official Vertex Pharmaceuticals Press Release (1/7/2025):
https://investors.vrtx.com/news-releases/news-release-details/vertex-announces-european-commission-approval-alyftrekr-new-once
