Health Canada has approved the use of Kalydeco (ivacaftor) in toddlers age 1-2 years with certain genetic mutations that prevent the correct functioning of the CFTR protein, the therapy’s developer announced.
With a pill taken twice a day, Kalydeco, by Vertex Pharmaceuticals, makes it possible to resolve the underlying cause of cystic fibrosis (CF) in people with “gating” mutations — those resulting in the production of a faulty CFTR protein that have its channel’s gate stuck closed.
The expansion of Kalydeco’s Market Authorization allows the use of this targeted therapy in young children with CF due to one of nine specific CFTR mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R.
“We believe it is important to treat the underlying cause of cystic fibrosis as early as possible, and are pleased that parents and physicians now have a medicine to treat the underlying cause of CF in indicated patients as young as one year of age,” Reshma Kewalramani, MD, said in a press release. Kewalramani is executive vice president and chief medical officer at Vertex.
“We look forward to working with the Canadian health officials and provinces to achieve rapid access to Kalydeco for this small, but important group of young children,” Kewalramani added.
The trial showed that after six months of treatment, the children had an almost three-fold reduction in mean levels of sweat chloride compared with those before therapy. The mean chloride levels dropped from 104.1 mmol/L to 33.8 mmol/L, which are considered within the range of healthy levels. (Mmol/L stands for millimole/liter, which is a metric measurement.
Kalydeco also improved the levels of several pancreatic biomarkers commonly used to determine CF-associated exocrine pancreatic dysfunction.
The safety profile of Kalydeco in this young population was similar to that reported in older children. Most adverse side effects reported were mild or moderate in severity, and no patient discontinued treatment due to side effects.
Vertex is still recruiting children younger than 24 months with CF due to CFTR gating mutations, to further explore the potential of Kalydeco in the ARRIVAL study.