Vertex Pharmaceuticals: Another Step Forward For Kalydeco

On February 21^st, Vertex Pharmaceuticals announces that the FDA approves a supplemental New Drug Application (sNDA) for orphan drug Kalydeco (Ivacaftor) for people with Cystic Fibrosis (CF), ages 6 and older, who have one of the 8 additional mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene :

•   G178R
•   S549N
•   S549R
•   G551S
•   G1244E
•   S1251N
•   S1255P
•   G1349D.

Kalydeco receives approval from the FDA in January 2012 for CF patients, ages 6 and older who have at least one copy of the G551D mutation. Thus, Kalydeco is currently approved for 9 mutations. The new approval affects approximately 150 in the United States.