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The Prescrire Awards for 2021

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Kaftrio° (ivacaftor + tezacaftor + elexacaftor): cited as Noteworthy at the 2021 Prescrire Drug Awards

In cystic fibrosis in patients aged 12 years and over with at least one F508del mutation in the CFTR gene > Learn more
 
 

Le Palmarès des Médicaments Drugs deemed Noteworthy provide a modest improvement in patient care.

• KAFTRIO° (ivacaftor + tezacaftor + elexacaftor) – Vertex Pharmaceuticals

In cystic fibrosis in patients aged 12 years and over with at least one F508del mutation in the CFTR gene (Prescrire Int n° 235).

Ivacaftor + tezacaftor + elexacaftor in cystic fibrosis with at least one F508del mutation: alleviation of respiratory symptoms, and sometimes fewer exacerbations.

Cystic fibrosis is a serious genetic disorder caused by mutations in the gene encoding the CFTR (cystic fibrosis transmembrane conductance regulator) protein. The most common mutation is the F508del (or deltaF508) mutation.

In patients with cystic fibrosis and at least one F508del mutation in the CFTR gene, triple therapy with the CFTR "modulators" ivacaftor + tezacaftor + elexacaftor (Kaftrio°) alleviated respiratory symptoms in four comparative trials, with a maximum duration of 24 weeks, in a total of 943 patients. Ivacaftor + tezacaftor + elexacaftor also reduced the incidence of exacerbations in heterozygous patients with one F508del mutation and a second mutation that results in minimal CFTR protein function.

The efficacy of ivacaftor + tezacaftor + elexacaftor triple therapy must be weighed against its harms, in particular upper respiratory tract infections, liver disorders, rash, muscle disorders and numerous drug interactions.

This triple therapy was deemed noteworthy mainly because of this demonstrated efficacy, albeit largely symptomatic and short-term. Any longer-term harms or possible effect on disease progression are as yet unknown.

A Pilule d'Or (Golden Pill) is granted to drugs that represent a major therapeutic advance in a field in which no treatment was previously available. Drugs included on the Honours List constitute a clear advance for some patients compared with existing therapeutic options, albeit with limitations. Drugs deemed "Noteworthy" provide a modest improvement in patient care.
SEE: > About the Prescrire Drug Awards for 2021

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> The Prescrire Drug Awards for 2021


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 FREE  "The Prescrire Awards for 2021" Prescrire International 2022; 31 (235): 79-82. (pdf, free)

 

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