We invest in scientific innovation to create transformative medicines for people with serious diseases. Our business is focused on developing and commercializing therapies for the treatment of cystic fibrosis, or CF, and advancing our research and development programs in other diseases.
Our goal is to develop treatment regimens that will provide benefits to all patients with CF and will enhance the benefits that currently are being provided to patients taking our medicines. Our marketed medicines are SYMDEKO/SYMKEVI (tezacaftor in combination with ivacaftor), ORKAMBI (lumacaftor in combination with ivacaftor) and KALYDECO (ivacaftor). These three medicines are collectively approved to treat approximately half of the 75,000 CF patients in North America, Europe and Australia.
We believe the triple combination regimens of next-generation correctors in combination with tezacaftor and ivacaftor that we are evaluating in Phase 3 clinical development could potentially provide benefits to all CF patients who have at least one F508del mutation in their cystic fibrosis transmembrane conductance regulator, or CFTR, gene (approximately 90% of all CF patients). This would provide (i) the first treatment for the underlying cause of CF for patients who have one copy of the F508del mutation in their CFTR gene and a second mutation that results in minimal CFTR function, whom we refer to as F508del/Min patients; and (ii) an improved treatment option for a majority of patients who are currently eligible for our products.