Elexacaftor/tezacaftor/ivacaftor
Elexacaftor/tezacaftor/ivacaftor, sold under the brand names Trikafta and Kaftrio, is a fixed-dose combination medication used to treat cystic fibrosis.
[8][12] Cystic fibrosis is an autosomal recessive genetic disorder of the CFTR protein which reduces chloride and sodium ion transport through the cell membrane, causing thicker than normal mucus secretions.
[19][20] Elexacaftor/tezacaftor/ivacaftor is a tridrug treatment in which the medications work together to increase the transport of chloride and sodium ions and correct fluid shifts that are dysregulated in cystic fibrosis.
[24] In the crystalline figure is shown ivacaftor, displayed as a gray ball and stick model on the bottom-right, bound to CFTR docked in a cleft formed by transmembrane helices at the protein-lipid interface.
The drug's manufacturer Vertex Pharmaceuticals will receive a rare pediatric disease priority review voucher for having developed this therapy.
[31] In March 2021, health regulators in Australia approved the combination for people aged 12 years and older with at least one copy of the F508del mutation.
[41] In December 2022, Pharmac announced it had reached a provisional agreement with Vertex funding Trikafta starting on 1 April 2023 for patients aged six or above.
[49] In March 2023, Ireland's Health Service Executive approved funding for the provision of Kaftrio to people aged six and over with cystic fibrosis.
[50][51] In November 2021, the Spanish government approved the reimbursement of the combination for people aged 12 years and older with at least one copy of the F508del mutation.
[52] In addition to Trikafta's high list price, Vertex has actively worked to prevent LMICs from accessing this drug combination.
This has led to groups of patients in three countries, namely India, Ukraine and South Africa, to initiate legal and regulatory actions to compel their governments to allow for the importation or local production of low-cost generic versions of Trikafta through compulsory licensing.
