Posted by Michael Wonder on 26 Sep 2020
FDA approves Kalydeco (ivacaftor) as first and only CFTR modulator to treat eligible infants with CF as early as four months of age
25 September 2020 - Approval provides opportunity to treat the underlying cause of cystic fibrosis earlier than ever before.
Vertex Pharmaceuticals today announced the U.S. FDA approved Kayldeco (ivacaftor) for use in children with cystic fibrosis ages four months to less than six months old who have at least one mutation in their cystic fibrosis transmembrane conductance regulator gene that is responsive to Kalydeco based on clinical and/or in vitro assay data.
Kalydeco is already approved in the U.S. and EU for the treatment of cystic fibrosis in patients ages six months and older.
