The recent debut of SYMDEKO and what it means for CF patients
The United States Food and Drug Administration has recently approved a combination therapy for this purpose that many believe will be the beginning of a reliance on triple-therapy regimens.
The combination they have approved is tezacaftor/ivacaftor and ivacaftor (SYMDEKO).
It has been approved for those who are over the age of twelve, and possess two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator, or CFTR, gene. Alternatively, they much have a minimum of one tezacaftor/ivacaftor-responsive mutation.
The combination therapy is produced by Vertex Pharmaceuticals, and is their third drug for cystic fibrosis patients to be approved within the United States. Many believe that this will "serve as clinical backbone for next-generation therapies that could treat up to 90% of patients with its wider range of efficacy than previous combination drugs."
Two phase 3 studies were involved in SYMDEKO's approval, and were responsible for testing the combination therapy in 750 patients who have cystic fibrosis, who are over the age of twelve, and have 2 copies of the F508del mutation in their CFTR genes.