European Commission Approves Kalydeco for Children as Young as Six Months

The European Commission has approved a label extension for Kalydeco (ivacaftor), allowing the therapy to be used in infants with cystic fibrosis (CF) who are between the ages of six months and one year.

The decision follows a positive opinion on Kalydeco, marketed by Vertex Pharmaceuticals, that was issued by the Committee for Medicinal Products for Human Use (CHMP) in October.

CF is caused by mutations in the gene CFTR (cystic fibrosis transmembrane conductance regulator), which encodes for a protein also named CFTR. When it functions properly, the CFTR protein acts like a gate on a cell’s membrane, allowing certain charged particles, or ions, to pass in and out.

Some CF mutations can cause the CFTR protein to “stick closed”; Kalydeco works by helping keep the ion “gate” open longer so that the protein can function properly.

In Europe, Kalydeco has been approved for people with CF, ages 12 months and older, who have at least one of nine specific mutations. These mutations are G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R.

In CF patients ages 18 and older, Kalydeco treatment is approved for an even greater number of mutations — 38 in total, including the R117H mutation.

The label extension now granted approves Kalydeco use in people with CF who are at least six months old, and have at least one of the nine mutations for which the treatment is approved among those under age 18. Children eligible for Kalydeco therapy also need to weigh at least 5 kg (about 11 pounds).

The approval for this younger patient population is based largely on the results of the Phase 3 clinical trial ARRIVAL (NCT02725567), which is evaluating Kalydeco in children younger than 2. The trial is still ongoing, but interim results have suggested that Kalydeco can be beneficial for this age group, with a safety profile in line with what has been seen in studies with older children and adults.

“Today’s approval is another milestone in our commitment to treat all people with CF as early in life as possible, given manifestations of CF are often present at birth,” Reshma Kewalramani, MD, executive vice president and chief medical officer of Vertex, said in a press release.

“For the first time, children with CF in Europe as young as 6 months with certain mutations now have access to a medicine that treats the underlying cause of their disease,” Kewalramani said.

The U.S. Food and Drug Administration in April 2019 approved Kalydeco‘s use in the United States as a treatment for children with CF ages 6 to 12 months.