FDA approves expanded use of three cystic fibrosis therapies
The FDA has approved expanded indications for three medications for the treatment of patients with cystic fibrosis with specific mutations in the CFTR gene, according to press release from Vertex Pharmaceuticals.
Use of elexacaftor/tezacaftor/ivacaftor (Trikafta) was expanded to include individuals aged 12 years and older with certain mutations in the CFTR gene that are responsive to the therapy based on in vitro data. Tezacaftor/ivacaftor (Symdeko) was also approved to include additional responsive mutations in individuals aged 6 years and older and ivacaftor (Kalydeco) was approved to include individuals aged 4 months and older, according to the release.
The new approvals allow more than 600 individuals with cystic fibrosis not previously eligible an opportunity to now receive these medications, according to the release. Previously, elexacaftor/tezacaftor/ivacaftor was approved for individuals with at least one F508del mutation and is now approved for 177 other mutations; tezacaftor/ivacaftor is approved for 127 additional mutations, for a total of 154 responsive mutations; and ivacaftor is approved for 59 additional mutations, for a total of 97 responsive mutations, according to the release. In addition, patients with cystic fibrosis who are eligible to receive ivacaftor are now eligible for tezacaftor/ivacaftor or elexacaftor/tezacaftor/ivacaftor, and those eligible for tezacaftor/ivacaftor will also be eligible for elexacaftor/tezacaftor/ivacaftor, according to the release.
“The approval for expanded use of three of our [cystic fibrosis] medicines based on our well-established in vitro model is a testament to the relentless commitment of our scientists to reach our goal of developing treatments for all people with [cystic fibrosis],” Reshma Kewalramani, MD, CEO and president of Vertex, said in the release. “We remain as committed today to reaching every patient who might benefit from our medicines as when we first started out on this journey 20 years ago, and this important milestone now enables hundreds of people with cystic fibrosis access to a treatment option to address the underlying cause of their disease — many for the first time.”