Vertex Pharmaceuticals announced it plans to request by year’s end that the label for Symdeko (tezacaftor/ivacaftor and ivacaftor), its recently approved disease-modifying therapy for cystic fibrosis (CF), be expanded to include children as young as 6 years old, the company announced in a recent investor report.
It also plans to request that Kalydeco (ivacaftor), its first treatment and one for CF patients with specific gating mutations, be approved to treat infants starting at age 6 months in both the U.S. and Europe.
Data from pivotal Phase 3 clinical trials on new triple combinations — expected to treat the 40 percent of patients who are not helped by the company’s existing CF portfolio — are also expected is be released this year and in early 2019.
If those results are positive, Vertex said that it also plans to file New Drug Applications with the U.S. Food and Drug Administration (FDA) requesting approval of these triple combinations — next-generation correctors called VX-659 and VX-445, each used in combination with tezacaftor and ivacaftor — by mid-2019.
Two Phase 3 studies are testing the VX-659 triple combo in CF patients 12 and older. One focuses on patients with one F508del mutation in the CFTR gene (the gene that is defective in CF) and one minimal function mutation (NCT03447249), and the other in patients with two copies of the F508del mutation (NCT03460990).
Both trials are fully enrolled, and outcomes are expected to be released by the close of this year.
Two other Phase 3 trials are evaluating the VX-445 triple combination in each of these same groups of patients — two F508del mutations (NCT03525548), and a F508del and minimal function mutation (NCT03525444).
Both these studies are still enrolling eligible patients across the U.S. (two F508 del mutations), and across the U.S. and elsewhere — Canada, Europe and Australia — for those with the two mutation types. Trial data is likely to be made public within the first quarter of next year.
At the recent 32nd North American Cystic Fibrosis Conference, Vertex presented data from Phase 2 clinical trials in each of these triple combinations, showing they can improve patients’ lung function.
Symdeko treats CF patients with two copies of the F508del mutation in the CFTR gene or with one mutation that responds to its combination — tezacaftor/ivacaftor — and was approved by the FDA for patients 12 and older in February. It is the company’s third FDA-approved treatment, after Kalydeco and Orkambi (lumacaftor/ivacaftor).
In a separate announcement on Thursday, Vertex said that Symdeko — under the brand name Symkevi — was approved in Europe to treat patients with two F508del mutations or one such mutation and one of 14 other minimal function mutations. This double combination was given a positive opinion by the Committee for Medicinal Products for Human Use (CHMP), a branch of the European Medicines Agency, in July.
The company also announced year-to-date revenue totaling $748 million from sales of Kalydeco, $947 million for Orkambi, and $475 million for Symdeco.