Approval is crucial for the future of cystic fibrosis treatment
Later, a larger study took place in which the tezacaftor/ivacaftor combination was compared to either ivacaftor monotherapy or simply placebo treatment groups, in around 250 cystic fibrosis patients. The timeframe of the evaluation was slightly over two months, and researchers were able to discover significant benefits associated with lung function offered to those who took the therapies as compared to those who took the placebo.
In the United States, oral ivacaftor is marketed as KALYDECO. It has capability of forcing CFTR proteins to remain at the cell surface for a prolonged amount of time, which aids in the process of salt and water transportation to the cell membrane. This process enables mucus to be cleared from the airways.
Jeffrey Leiden, MD, PhD, is the chairman, president, and CEO of Vertex. He comments on why SYMDEKO's approval by the United States Food and Drug Administration is so crucial, "this approval is an important milestone in our journey to treat every person with CF, and we remain committed to urgently advancing our efforts to develop new medicines that treat the underlying cause of CF for the many people still waiting."