Approval was based on data from two phase 3 studies, EVOLVE and EXPAND, that enrolled 744 people with CF ages 12 and older with two copies of the F508del mutation or with one F508del mutation and a second mutation predicted to be responsive to tezacaftor/ivacaftor.
Across both studies, patients treated with Symdeko experienced statistically significant improvements in lung function. The treatment was generally well tolerated; the most common adverse reactions were headache and nasopharyngitis.
Results from an interim analysis of the ongoing, 96-week EXTEND Phase 3 rollover study add to the growing body of evidence supporting the benefit of long-term treatment of the underlying cause of the disease. Initial improvements in lung function observed in the EVOLVE study were sustained for up to 48 weeks.
Symdeko is a combination of tezacaftor and ivacaftor. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface where ivacaftor can increase the amount of time the protein stays open.
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases.
In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.
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