Kalydeco Open to Infants in Europe Starting at 4 Months Old

The European Commission has approved Kalydeco (ivacaftor), by Vertex Pharmaceuticals, to treat infants as young as four months old with cystic fibrosis (CF) caused by certain mutations.

The decision to extend Kalydeco’s label to younger babies, weighing at least 5 kg (about 11 lbs) and with relevant mutations, follows a recommendation by the Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency (EMA).

“Today’s approval is a testament to our commitment to keep going until all people with CF have a treatment option,” Reshma Kewalramani, MD, Vertex’s CEO and president, said in a press release.

CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which encodes the CFTR protein. This protein normally functions like a “gate” on the surface of cells that can open and close to control the movement of charged salts (ions). The mutated protein in people with CF does not work correctly.

Kalydeco is a CFTR modulator, meaning it works to correct the underlying defect in the CFTR protein. Specifically, Kalydeco is a potentiator of the CFTR protein: it helps to “open the gate” when specific mutations cause it to be “stuck closed.” This makes it easier to transport salts and water in and out of cells, thereby easing disease symptoms. 

Kalydeco was previously approved in Europe for people with CF, ages 6 months and older, who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, R117H, S1251N, S1255P, S549N or S549R. The new approval covers younger infants with these mutations.

“Our very first CFTR modulator, Kalydeco, was first approved eight years ago, for certain CF patients ages 6 years and older. With today’s approval, babies as young as 4 months are eligible and we believe early treatment is important in managing CF,” Kewalramani said.

Kalydeco was also recently approved in the U.S. for infants with these CF gating mutations as young as 4 months old.

These approvals were based on data from the ongoing Phase 3 ARRIVAL clinical trial (NCT02725567), sponsored by Vertex, that included six infants between the ages of 4 and 6 months who were treated with Kalydeco. Safety findings in these infants were consistent with those reported in older age groups.

ARRIVAL, a 24-week safety trial in which all enrolled will be treated, is still recruiting infants and toddlers up to 24 months old at sites across the U.S., and in Australia, Canada, Ireland and the U.K. Information is available here.

Kalydeco is also now available to eligible patients in Germany, Vertex said in its release, and will soon be available in other European countries with established long-term reimbursement agreements with the company, including U.K., the Republic of Ireland, and Denmark.

Vertex is working with relevant authorities in other European countries to allow access to the therapy, it stated.