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FDA Approves Use of CF Drug in Younger Children

Vertex news release; 2018 Aug 15

Vertex Pharmaceuticals Incorporated recently announced that the US Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) to include use in children with cystic fibrosis (CF) aged 12 to <24 months who have at least 1 mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to Kalydeco based on clinical and/or in vitro assay data. In the US, Vertex Pharmaceuticals Incorporated is headquartered in Boston.

Dosage/administration: For pediatric patients aged 12 months to <6 years of age and weighing 7 kg to <14 kg: 1 50 mg packet mixed with 1 teaspoon (5 mL) of soft food or liquid and administered orally every 12 hours with fat-containing food. For pediatric patients aged 12 months to <6 years of age and ≥14 kg: 1 75 mg packet mixed with 1 teaspoon (5 mL) of soft food or liquid and administered orally every 12 hours with fat-containing food. Kalydeco is not recommended for pediatric patients aged <12 months.

Adverse effects: The most common adverse drug reactions to Kalydeco (occurring in

≥8% of patients with CF who have a G551D mutation in the CFTR gene) were headache, oropharyngeal pain, upper respiratory tract infection, nasal congestion, abdominal pain, nasopharyngitis, diarrhea, rash, nausea, and dizziness.

Citation:

FDA approves Kalydeco (ivacaftor) as first and only medicine to treat the underlying cause of CF in children ages 12 to <24 months with certain mutations in the CFTR gene. [news release]. Boston, MA: Vertex Pharmaceuticals Incorporated. August 15, 2018. https://investors.vrtx.com/news-releases/news-release-details/fda-approves-kalydecor-ivacaftor-first-and-only-medicine-treat. Accessed August 30, 2018.