Dive Brief:
- The Food and Drug Administration has given the nod to Vertex Pharmaceuticals to extend use for Kalydeco to infants aged 12 to under 24 months with at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene.
- The approval is based on data from the ongoing Phase 3 open-label ARRIVAL safety study in this age group. The study showed safety results similar to previous studies in older children and adults, as well as drops in their mean sweat chloride levels.
- Kalydeco is the first drug treating the underlying cause of cystic fibrosis in children of this age with at least one mutation in the CFTR gene.
Dive Insight:
While cystic fibrosis is rare, it does affect roughly 75,000 people in the U.S. It has no cure, and until relatively recently, treatment was purely symptomatic.
In 2012, Vertex's Kalydeco (ivacaftor) became the first FDA-approved drug to treat the underlying cause of cystic fibrosis in people with the disorder aged 6 and older who have at least one copy of the G551D mutation. It was also one of the fastest FDA approvals ever, at around three months.
Since its first launch, Kalydeco's label has been extended to cover children and adults aged 2 years and older who have one of 38 ivacaftor-responsive mutations in the CFTR gene, as well as in the subgroup of patients with one of five residual functional mutations.
Today's news brings the age limit down to one year, and allows earlier treatment, which could improve long-term outcomes.
"This the first regulatory approval of a CFTR modulator in this age group," said Margaret Rosenfeld, a pediatrics professor at the University of Washington School of Medicine. "The premise of newborn screening for CF is to intervene very early in the course of disease with the goal of improving long term outcomes, so this is a significant milestone for parents and caregivers of young children with CF."
As its label has expanded, Kalydeco's product revenues have grown, from $189.6 million in the second quarter of 2017 to $253.1 million in the second quarter of 2018.
In the same period, Orkambi (ivacaftor/lumacaftor) product revenues fell slightly from $324.4 million to $311.3 million, partly driven by sales moving to the newly-launched Symdeko (tezacaftor/ivacaftor and ivacaftor). Orkambi's recent label extension to children aged two to five years who have two copies of the F508del mutation may increase the product's sales once more.
Despite its rising product revenues, Vertex has been hit with pushback because of cost.
The U.K.'s National Institute for Health and Care Excellence (NICE) suspended review of Symkevi (marketed as Symdeko in the U.S.) last week after the company and the health technology assessment body could not agree on a price for the drug that both sides saw as acceptable.
The Institute for Clinical and Economic Review (ICER), an independent group that evaluates the economics of prescription medications, also views the prices of Vertex's drugs as too high, stating that the list prices of Vertex's Kalydeco, Orkambi and Symdeko would have to come down between 71% and 77% to meet common cost-effectiveness thresholds.