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FDA Expands Labeling for Kalydeco (Ivacaftor) to Include Use in Cystic Fibrosis Patients 6 Years of Age and Older With R117H Mutation

Approximately 500 people in U.S. have disease subtype

The FDA has approved a supplemental new drug application (sNDA) for the use of Kalydeco (ivacaftor, Vertex Pharmaceuticals) in patients aged 6 years and older with cystic fibrosis (CF) who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The approval follows a recommendation by the FDA’s Pulmonary and Allergy Drugs Advisory Committee to approve the medication for use in this subpopulation. Kalydeco is now approved for use in the U.S. in subjects aged 6 years and older with CF with one of the following ten mutations: R117H, G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. The new approval is based on data from a phase III study of ivacaftor that enrolled 69 subjects aged 6 years and older with CF and the R117H mutation.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. In people with the R117H mutation, the CFTR protein reaches the cell surface but does not function properly. Approximately 500 people aged 6 years and older have this mutation in the U.S. With the new approval, Kalydeco is now indicated for the treatment of more than 3,100 people aged 6 years and older in North America, Europe, and Australia who have specific mutations in the CFTR gene.

Kalydeco (ivacaftor) is the first medication to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, Kalydeco is an oral medication designed to keep CFTR proteins at the cell surface open more often to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

Kalydeco is approved in the U.S., Europe, Canada, Switzerland, Australia, and New Zealand to treat people aged 6 years and older with CF with specific genetic mutations in the CFTR gene. Only in the U.S. is the drug approved to treat people with CF who have the R117H mutation.

Kalydeco is not effective in patients with CF with two copies of the F508del mutation (F508del/F508del) in the CFTR gene. The safety and efficacy of the drug in children with CF younger than 6 years of age have not been established.

Ivacaftor can cause serious adverse reactions, including abdominal pain and high liver enzymes in the blood. The most common adverse effects associated with the use of ivacaftor include headache; upper respiratory-tract infection (i.e., the common cold), including sore throat, nasal or sinus congestion, and runny nose; abdominal pain; diarrhea; rash; and dizziness.

CF is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe, and Australia. The median predicted age of survival for a person with CF is between 34 and 47 years, but the median age of death remains in the mid-20s.

Source: Vertex Pharmaceuticals; December 29, 2014.

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