- Vertex Pharmaceuticals Incorporated upped its annual sales forecast for its older cystic fibrosis (CF) mainstay Kalydeco by $30 million to between $770 million and $800 million, reflecting a label expansion granted by the Food and Drug Administration on August 1.
- With the new approval, Kalydeco is now OK'd for use in an estimated 600 additional patients who have one of five residual functional mutations that damages the crucial CFTR gene.
- Vertex had previously raised its guidance for Kalydeco to between $740 and $770 million following an earlier label expansion — notable particularly because that approval came from analyses of in vitro data and real-world clinical data.
Shares in Vertex have risen in value by 106% this year, spurred by investor optimism in the biotech's pipeline advances and a strengthening position for its two on-market drugs, Kalydeco (ivacaftor) and Orkambi (lumacaftor/ivacaftor).
Positive results from two Phase 3 studies testing a combination of Kalydeco with a second-generation corrector known as tezacaftor were a major catalyst behind that doubling in value. The success with the teza/iva combo, as it's known, helped to shake off lingering concerns with slower-than-expected sales from Orkambi.
In one of the two trials, the combo significantly improved lung function in patients with two copies of a mutation called F508del — potentially indicating superior efficacy than even Orkambi with fewer respiratory adverse events.
The other study, known as EXPAND, looked at patients with one copy of that F508del mutation and another mutation that results in residual function of a protein encoded by the CFTR gene. As part of its design, EXPAND randomized some patients to treatment with Kayldeco monotherapy before switching to combination treatment.
Data from that monotherapy arm supported Tuesday's approval for patients with five residual function mutations.
In addition to tez/iva, Vertex is also developing three triple combinations that it hopes could expand the number of patients it can treat to about 90% of all CF patients. Vertex's currently marketed therapies reach about 50% of the total patient population according to analysts.