The FDA approves ivacaftor for the treatment of infants with cystic fibrosis as early as 6 months of age

Vertex Pharmaceuticals has announced that the FDA has approved the use of ivacaftor in children with cystic fibrosis aged 6 to less than 12 months of age and presenting with at least one mutation in their transmembrane conductance regulator gene of the Cystic fibrosis that is sensitive to ivacaftor according to clinical criteria or in vitro data badysis.

Ivacaftor (Kalydeco) is already approved in the United States, Canada and the European Union for the treatment of cystic fibrosis in children aged at least 12 months, according to a press release of the 39; company.

The new approval in infants was based on data from an open cohort of 24 weeks of phase 3 (ARRIVAL) of 11 children with cystic fibrosis aged 6 to less than 12 months who had one of the 10 mutations: CFTR gene (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H). In the ARRIVAL cohort, ivacaftor treatment in this population showed a safety profile similar to that observed in previous phase 3 studies in older children and adults, the release said. Most side effects were mild or moderate and the most common effects were cough (64%), nasal congestion (36%) and rhinorrhea (36%). ARRIVAL data also showed a reduction in mean sweat chloride after 24 weeks of treatment. Of the six infants with matched sweat chloride samples at baseline and at 24 weeks, the mean absolute change was -58.6 mmol / L, according to the release.

Ivacaftor is an oral treatment that keeps CFTR proteins on the surface of cells longer open, to improve the transport of salt and water across the cell membrane, which improves hydration and the elimination of mucus from the airways. This treatment is available as pills of 25 mg, 50 mg and 75 mg in pediatric patients from 6 months to less than 6 years old. In adults and children aged 6 years and older, ivacaftor is available as 150 mg tablets, according to the release.

"[The] approval for Kalydeco allows doctors to start treating the underlying cause of [cystic fibrosis] in eligible infants from the age of 6 months for the first time, with the potential to alter the course of the disease ", Margaret Rosenfeld, MD, MPH, Seattle Children's Hospital's badistant physician and a professor in the pediatric department of the University of Washington's School of Medicine, the statement said.

Disclosure: Rosenfeld was one of the principal investigators of the ARRIVAL study, sponsored by Vertex Pharmaceuticals.