CHMP Favors Expanding Kalydeco Label to Include Young Children With R117H Mutation

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion for a label extension of the cystic fibrosis (CF) treatment Kalydeco (ivacaftor).

If approved by the European Commission, the extension would allow Kalydeco use in patients 6 months and older who weigh at least five kg (about 11 lb) and have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (the gene defective in CF).

Kalydeco, developed and marketed by Vertex Pharmaceuticals, is a CFTR potentiator. The protein encoded by the CFTR gene (CFTR protein) works like a gate on the membrane of cells, allowing water molecules and chloride ions to pass in and out of the cell. Some CF-causing mutations, including R117H, cause this gate to become “stuck” in the closed position. Conceptually, CFTR potentiators such as Kalydeco work by helping to keep the gate open.

In Europe, Kalydeco is currently approved for people with CF, ages 6 months and older, who have at least one of nine mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. It is also approved for CF patients 18 and older with the R117H mutation.

If the European Commission approves the new label extension, Kalydeco would become the first medicine approved for children with CF caused by the R117H mutation.

“Today’s announcement is important for young people with CF, as early intervention and treatment of this devastating and progressive disease is key to keeping patients healthier longer,” Carmen Bozic, MD, Vertex’s executive vice president and chief medical officer, said in a press release. “This milestone also brings us one step closer to achieving our ultimate goal of bringing medicines forward to all people with CF.”

According to Vertex, if approved, Kalydeco will become available to patients shortly after it receives marketing authorization in European countries where long-term reimbursement agreements have already been made. The company will work closely with relevant authorities in EU countries to secure access for patients.

In the United States, Kalydeco is currently approved for use in CF patients 6 months and older, including those with the R117H mutation.