FDA Approves Symdeko to Treat the Underlying Cause of Cystic Fibrosis
Drug maker Vertex has announced that the FDA has approved tezacaftor/ivacaftor and ivacaftor (Symdeko) for the treatment of the underlying cause of cystic fibrosis (CF) in patients aged 12 or older who have 2 copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene, or who have at least 1 genetic mutation that is responsive to treatment with tezacaftor/ivacaftor.
Drug maker Vertex has announced that the FDA has
CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. There are approximately 2000 known mutations of the gene, some of which result in a CFTR protein that presents with abnormal folding within the cell, which prevents it from reaching the cell surface. The newly approved drug works by using tezacaftor to address the trafficking and processing defect of the CFTR protein to allow it to reach the cell surface, where ivacaftor can increase the amount of time that the protein stays open.
“Today is an exciting day for the CF community. The approval of Symdeko, our third disease-modifying CF medicine, offers many patients an important new treatment option,” said Jeffrey Leiden, MD, PhD, Vertex's chairman, president, and CEO, in a statement.
Currently approved
Vertex is also in the
Both combinations, says Vertex, were generally well tolerated in phase 2 studies, with a low rate of discontinuation due to adverse events.
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