Trikafta (Triple Combination Therapy: Elexacaftor/Tezacaftor/Ivacaftor) Is Only Effective in CF Patients With f508del Gene Mutation
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It helps the protein made by the CFTR gene mutation function more effectively. Currently available therapies that target the defective protein are treatment options for some patients with cystic fibrosis, but many patients have mutations that are ineligible for treatment. Trikafta is the first approved treatment that is effective for cystic fibrosis patients 12 years and older with at least one F508del mutation, which affects 90% of the population with cystic fibrosis or roughly 27,000 people in the United States.
People with two copies of the F508del mutation had a 10 percent increase in lung function compared to treatment with the modulator tezacaftor/ivacaftor (Symdeko®), and people with one copy of F508del had more than a 14 percent increase in lung function compared to placebo. In people with one copy of the F508del mutation, Trikafta™ was also associated with significant improvements in sweat chloride, pulmonary exacerbations and quality of life. A study of Trikafta™ in children with CF ages 6-11 years old is currently underway.
Many mutations, however, cause multiple defects [Veit G, Avramescu RG, Chiang AN].
Translation of these potentiator combinations to the clinic should now be a priority.
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