Access to once-a-day triple therapy moves a step closer

Today 7 March, the Medicines and Healthcare products Regulatory Agency (MHRA) has approved the new triple modulator drug, Alyftrek (deutivacaftor/tezacaftor/vanzacaftor), saying it is safe and effective for eligible people with 1 or more F508del mutations or another mutation in the CFTR gene that is responsive to Alyftrek, in people 6 years and over. 

The next stage of the process is the ongoing assessment by the National Institute for Health and Care Excellence (NICE) who will make a decision on whether it should be made available on the NHS. We are hopeful that this will be completed during the summer. 

Alyftrek is a new once-a-day triple combination modulator drug that will be an alternative to Kaftrio. Two clinical trials show that the drug was found to be as effective as Kaftrio in improving lung function, and more effective at reducing sweat chloride levels. It is also potentially a treatment option for people with a small number of gene mutations not covered by the license for Kaftrio.

Click here to find out more about Alfytrek (vanza triple) and read our FAQs

What happens next?

Alyftrek was approved in the US by the Food and Drug Administration (FDA) in December, and approval from the MHRA ensures the medicine is safe. We now are waiting to hear from the National Institute for Health and Care Excellence (NICE) about the medicine’s clinical and cost effectiveness and the Trust has submitted evidence as part of this. 

This NICE assessment is separate from last year’s appraisal of Kaftrio, Symkevi and Orkambi, and the process is more streamlined so we are expecting this to conclude this summer.

Access to Kaftrio is not impacted by this announcement. 

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.”

Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex, said “For more than 20 years we have been focused on discovering medicines that treat the underlying cause of the disease with the goal of helping people live longer and better lives. The approval of ALYFTREK, our fifth CFTR modulator regimen, represents another significant milestone in that journey for people with CF in the UK”