FDA Expands Use of Three Cystic Fibrosis Medications

The Food and Drug Administration (FDA) has expanded the approval of Trikafta® (elexacaftor/tezacaftor/ivacaftor and ivacaftor; Vertex), Symdeko® (tezacaftor/ivacaftor and ivacaftor; Vertex), and Kalydeco® (ivacaftor; Vertex) to include use in cystic fibrosis patients with additional cystic fibrosis transmembrane conductance regulator (CFTR) gene mutations.

Trikafta is indicated for patients aged 12 years and older who have at least 1 F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive based on in vitro data. The expanded approval now includes 177 additional mutations. Symdeko is indicated for patients aged 6 years and older who are homozygous for the F508del mutation or have at least 1 mutation in the CFTR gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence. The expanded approval now includes 127 additional mutations, for a total of 154 Symdeko-responsive mutations. Kalydeco is indicated for patients aged 4 months and older who have 1 mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data. The expanded approval now includes an additional 59 mutations, for a total of 97 Kalydeco-responsive mutations.

According to the Company, these approvals allow for more than 600 patients with rare cystic fibrosis mutations to now be eligible to receive treatment. Moreover, the approval allows patients who are eligible for Kalydeco to also be eligible for Symdeko or Trikafta; those eligible for Symdeko are now eligible for Trikafta as well.

The full list of CFTR gene mutations for Trikafta, Symdeko and Kalydeco can be found in the updated prescribing information for each respective product.

For more information visit vrtx.com.

Reference

Vertex announces FDA approvals of Trikafta® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko® (tezacaftor/ivacaftor and ivacaftor) and Kalydeco® (ivacaftor) for use in people with CF with certain rare mutations. [press release]. Boston, MA: Vertex Pharmaceuticals; December 21, 2020.