The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion regarding the use of Kalydeco (ivacaftor) in infants ages 6 months to a year with cystic fibrosis (CF).
CF is caused by mutations in the gene CFTR (cystic fibrosis transmembrane conductance regulator), which encodes for a protein of the same name that acts as a sort of gate, an ion channel, allowing certain salt ions to flow in and out of cells. Some CFTR-causing mutations cause this gate to become stuck closed; Kalydeco can help these stay open longer.
Kalydeco currently is approved in Europe for the treatment of people with CF with these mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. But its approval is limited to those who are at least a year old. It is similarly approved in the United States, Canada, Australia, and New Zealand.
The positive CHMP opinion is based largely on results from the Phase 3 clinical trial ARRIVAL (NCT02725567), which is evaluating the use of Kalydeco in children under age 2. The trial is still in progress, but interim results have shown that Kalydeco has the potential to modify the course of toddlers with CF.
If EMA adopts the new CHMP opinion, Kalydeco would be approved for use in children with CF who are as young as six months of age, and who have one of the CF-causing mutations mentioned above.
Kalydeco is also approved by EMA for the treatment of people older than 18 who have the CF-causing mutation R117H; this mutation is not included in the CHMP opinion.
“Cystic fibrosis is present from birth and symptoms frequently manifest in infancy, which is why it is so important to treat the condition as early as possible,” Reshma Kewalramani, MD, executive vice president and chief medical officer at Vertex, said in a press release. “We are committed to treating every person with this life-limiting disease, and today brings us one step closer to providing a medicine for very young children with cystic fibrosis.”