Triple combination medicine deutivacaftor/tezacaftor/vanzacaftor approved for cystic fibrosis
Source: gov.uk (2025-03-07)

As with all products, the MHRA will keep its safety under close review

The Medicines and Healthcare products Regulatory Agency (MHRA) has approved the triple combination medicine deutivacaftor/tezacaftor/vanzacaftor (brand name Alyftrek) to treat cystic fibrosis (CF) in people aged six years and older who have specific mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that have been shown in trials to respond to the therapy. This includes F508del, which is the most common cystic fibrosis causing mutation.

Cystic fibrosis is an inherited condition caused by a faulty CFTR gene, which helps regulate the flow of water and chloride in and out of the lungs and other organs. This causes sticky mucus to build up in the lungs and digestive system, which can lead to lung infections and problems with digesting food.

Deutivacaftor/tezacaftor/vanzacaftor is a CFTR “modulator”, meaning it is designed to correct the malfunctioning protein made by the CFTR gene in people with cystic fibrosis.

Julian Beach, MHRA Interim Executive Director, Healthcare Quality and Access, said:

“Keeping patients safe and enabling their access to high quality, safe and effective medical products are key priorities for us.

“We confirm that the appropriate regulatory standards for the approval of this medicine have been met.

“As with all products, we will keep its safety under close review.”

David Ramsden, Cystic Fibrosis Trust Chief Executive, said:

“Today’s MHRA approval is another important step in making sure as many people with CF as possible can benefit from the best available treatments.

“We now hope that NICE will move quickly to complete its assessment of the medicine to enable it to be prescribed on the NHS.

“Today is good news, but we never forget that these medicines are not a cure, and do not work for some people. Cystic Fibrosis Trust will not stop until everyone with CF can live a life that’s not limited by their condition.”

Deutivacaftor/tezacaftor/vanzacaftor is administered as a tablet once a day.

Food or drink containing grapefruit should be avoided during treatment.

In two randomised phase 3 studies involving 480 participants aged 12 years and over (studies 121-102 and 121-103), deutivacaftor/tezacaftor/vanzacaftor was found to be as effective at improving lung function as ivacaftor/tezacaftor/elexacaftor (Kaftrio), a currently approved triple combination therapy, and more effective at reducing sweat chloride levels. These findings are supported by additional data from an open‑label, phase 3 study (study 121-105, Cohort B1).

The most common side effects in the clinical trials were headache (15.8%) and diarrhoea (12.1%). For the full list of all side effects reported with this medicine, see Section 4 of the Patient Information Leaflet (PIL) or the Summary of Product Characteristics (SmPC) available on the MHRA website. (https://products.mhra.gov.uk/)

As with any medicine, the MHRA will keep the safety and effectiveness of deutivacaftor/tezacaftor/vanzacaftor under close review. Anyone who suspects they are having a side effect from this medicine are encouraged to talk to their doctor, pharmacist or nurse and report it directly to the Yellow Card scheme, either through the website (https://yellowcard.mhra.gov.uk/) or by searching the Google Play or Apple App stores for MHRA Yellow Card.

Notes to editors

The new marketing authorisation was granted via a national route (https://www.gov.uk/government/collections/licencing-how-to-apply) on 7 March 2025 to Vertex Pharmaceuticals (Europe) Limited.

More information can be found in the PIL and SmPC which will be published on the MHRA Products website (https://products.mhra.gov.uk/) within 7 days of approval.

For more information about cystic fibrosis, visit here (https://www.nhs.uk/conditions/cystic-fibrosis/#:~:text=Cystic%20fibrosis%20is%20a%20genetic,the%20lungs%20and%20digestive%20system.).

The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe. All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks.

The MHRA is an executive agency of the Department of Health and Social Care.

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