Canada
In June 2020, Health Canada approved Trikafta for patients ages 12 and up. In September 2021, the provinces Alberta and Saskatchewan announced they will join Ontario in funding the medication. They will determine coverage on a case-by-case basis using criteria that has not yet been announced. European Union
In June 2020, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended its approval for the treatment of cystic fibrosis. It was approved for medical use in the European Union in August 2020. Norway
On 25 April 2022, Beslutningsforum for nye metoder approved Kaftrio for treatment of cystic fibrosis. New Zealand
In February 2022, Pharmac recommended, with medium priority, funding for patients age 12 and above. Spain
In November 2021, the Spanish government approved the reimbursement of Kaftrio for patients ages 12 and older with at least one copy of the F508del mutation. Economics
United States
The list price of a years treatment in the US is US$311,000. However, a 2020 report by Institute for Clinical and Economic Review found that the price has made the treatment not cost effective and that "an appropriate health-benefit price would range from $67,900–$85,500 per year". Australia
Following the listing of Trikafta on the Pharmaceutical Benefits Scheme in 2022, the cost for CF patients 12 years or older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene is $42.50 per month, or $6.80 for concession card holders.
Research
CFTR mutations that are responsive to elexacaftor/tezacaftor/ivacaftor were determined by an in-vitro study of Fischer Rat Thyroid (FRT) cells that expressed mutant CFTR. Elexacaftor/tezacaftor/ivacaftor showed effectiveness with mutations where the CFTR protein was being successfully delivered to the cell surface. References
External links
"Elexacaftor". Drug Information Portal. U.S. National Library of Medicine. "Ivacaftor regimen with Tezacaftor". Drug Information Portal. U.S. National Library of Medicine.