Summary
Vertex Pharmaceuticals announced a significant development on February 12, 2018, with the U.S. Food and Drug Administration's (FDA) approval of SYMDEKO™ (tezacaftor/ivacaftor and ivacaftor). This new treatment is approved for cystic fibrosis (CF) patients aged twelve and older who have specific genetic mutations (two copies of the F508del mutation or at least one mutation responsive to tezacaftor/ivacaftor). The company has set a wholesale acquisition cost of $292,000 annually for SYMDEKO in the U.S. Vertex also provided updated financial guidance for the full year 2018, projecting total CF net product revenues between $2.65 billion and $2.80 billion. This growth is expected to be driven by an increased patient base across their CF portfolio, including the U.S. launch of SYMDEKO and wider adoption of ORKAMBI outside the U.S. The company reiterated its previously announced expense guidance for 2018, with combined GAAP R&D and SG&A expenses projected between $1.80 billion and $1.95 billion, and non-GAAP expenses between $1.50 billion and $1.55 billion.
Key Highlights
- 1FDA approval of SYMDEKO™ (tezacaftor/ivacaftor and ivacaftor) for a specific subset of cystic fibrosis patients aged 12 and older.
- 2SYMDEKO™ targets patients with two copies of the F508del mutation or at least one mutation responsive to tezacaftor/ivacaftor.
- 3Annual wholesale acquisition cost for SYMDEKO™ in the U.S. is set at $292,000.
- 4Full-year 2018 total CF net product revenue guidance reiterated at $2.65 billion to $2.80 billion.
- 5Revenue growth expected from increased patient uptake of Vertex's CF medicines, including the U.S. launch of SYMDEKO™ and increased ORKAMBI use internationally.
- 6Reiteration of 2018 combined GAAP R&D and SG&A expenses guidance ($1.80B - $1.95B) and non-GAAP guidance ($1.50B - $1.55B).