The Welsh government and Vertex Pharmaceuticals have reached an agreement that will allow access to the company’s Kaftrio (ivacaftor/tezacaftor/elexacaftor) for cystic fibrosis (CF) patients as soon as the medication is approved in Europe.
Kaftrio is a triple combination therapy for the treatment of CF, designed to be used in a combination regimen with Kalydeco, also marketed by Vertex. A similar triple combo has already been approved in the U.S. under the name Trikafta.
In Europe, the Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, issued a positive opinion in June; the European Commission will make a final decision on marketing authorization.
“News of a deal between the Welsh Government and Vertex Pharmaceuticals is fantastic,” David Ramsden, chief executive of the U.K.’s Cystic Fibrosis Trust, said in a press release. “While this is a great moment, we will not stop until everyone with cystic fibrosis across the UK can truly live a life unlimited.”
Under the agreement, CF patients living in Wales ages 12 and older who have either two copies of the F508del mutation (the most common CF-causing mutation), or one F508del mutation and one minimal function mutation in the CFTR gene (the gene defective in CF) will be eligible for Kaftrio treatment.
Patients who fall outside these criteria, namely those with rare mutations, may still benefit from Vertex’s treatments when deemed appropriate by their clinicians.
CHMP’s positive opinion was based on the results of two Phase 3 clinical trials, AURORA F/MF (NCT03525444) and AURORA F/F (NCT03525548). The triple combination significantly improved lung function and lessened pulmonary exacerbations in people with CF who have either two F508del mutations (F/F) or one F508del and one minimal function mutation (F/MF).
“This is life changing for us and for so many other people. To know that our daughter can now have a future is completely overwhelming. We’re beyond happy.” said Rebecka Bow, the mother of a daughter, Sofia, with CF.
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