- Cystic fibrosis-focused Vertex Pharmaceuticals on Wednesday secured another approval from the Food and Drug Administration for its key drug Kalydeco (ivacaftor), expanding the drug's label for use in roughly 900 people ages 2 and older who have one of 23 residual function mutations in the CFTR gene.
- Based on the approval, Vertex is bumping up its sales guidance for Kalydeco in 2017, now forecasting between $740 million to $770 million in sales, up from the previous estimate of $710 million to $730 million.
- Shares in Vertex have moved steadily higher since March, when the biotech announced strong data for an experimental cystic fibrosis therapy combining Kaldeco with a second-generation corrector called tezacaftor. Vertex plans to submit for approval in the third quarter.
Boston-based Vertex Pharmaceuticals is almost exclusively dependent on its portfolio of cystic fibrosis therapeutics, which together brought in nearly $1.7 billion in sales last year.
But slower-than-expected sales for its newer combination drug Orkambi (lumacaftor/ivacaftor) had led the company to stake out conservative 2017 guidance at the start of the year. Sales growth for Orkambi strengthened in the first quarter, and Wednesday's new approval should expand the market for Kalydeco — giving Vertex more momentum.
Vertex's new guidance would represent between 5.2% and 9.5% growth over the $703 million Kalydeco brought in last year.
Interestingly, Kalydeco's new approval came from analyses of in vitro data and five years of real-world clinical data. Kalydeco is now approved to treat CF patients who have one of 33 mutations in the CFTR gene.
The biotech has a raft of trials designed to expand indications for both drugs. Most important are the EVOLVE and EXPAND studies looking at Kalydeco plus tezacaftor, which read out positive in March.
The larger of the two, EVOLVE, tested the combo in CF patients aged 12 or older with two copies of the F508del mutations, while EXPAND studied the two-drug pairing in patients with one mutation that results in residual CFTR function and one F508del mutation.
A Phase 3 study of the combination is under way in people with one copy of the F508del mutation and a second mutation that results in a gating defect, due to complete enrollment in the first half of 2017. Vertex is also carrying out a Phase 3 trial in six to 11 year old children with these mutations.
Orkambi, on the other hand, is currently being assessed for use in children aged two to five with two copies of the F508del mutation.