Originally posted on Orphan Druganaut Blog:
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On February 21st, 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.
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