FDAnews
www.fdanews.com/articles/170507-fda-widens-kalydecos-age-range-to-encompass-very-young-children

FDA Widens Kalydeco’s Age Range to Encompass Very Young Children

March 25, 2015

Hundreds of children as young as two years old will now be able to get Vertex Pharmaceuticals’ game-changing cystic fibrosis treatment, following FDA approval last week.

Kalydeco’s (ivacaftor) expanded indication includes children between the ages of two and five who have one of 10 mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R or R117H. The drug is also available in a new granulated formulated that can be mixed into soft foods or liquids for young patients who can’t yet swallow tablets.

Kalydeco was originally approved in the U.S. in 2012 for patients six years and older who have at least one copy of the G551D mutation. The FDA has since expanded the indication for that age group to include nine other mutations. Last week’s approval is the first indication for children younger than six.

The largest CF patient group — those with the Fdel508 mutation — may soon be getting good news as well. In November, Vertex submitted applications to the FDA and European Medicines Agency for Kalydeco in combination with its experimental CF drug lumacaftor for patients with Fdel508.

Prior to 2012, the only options for CF patients were maintenance therapies for thinning mucus in the airways or treating lung infections. Kalydeco is the first and only drug that corrects disease-causing defects in the CFTR protein. — Lena Freund