Cystic fibrosis modulator therapies help to correct the malfunctioning cystic fibrosis conductance regulator (CFTR) protein, which is made by the CFTR gene.
CFTR modulator therapies are also known as precision medicines. Because CF is caused by over 2,000 known genetic mutations only certain modulator therapies help people with CF with specific mutations.
CF is caused by a mutation in the gene that produces the cystic fibrosis transmembrane conductance regulator (CFTR) protein.
Proteins are the building blocks of our body, and the CFTR protein controls the flow of salt and fluid in and out of our cells in different parts of our body.
Mutations in the CFTR gene cause the CFTR protein to not work properly, or not be made at all, which causes mucus to become thick and sticky.
Find out more about the CFTR protein
New medications known as CFTR modulators have been developed that work on treating the underlying cause of CF by helping to correct the malfunctioning protein made by the CFTR gene.
Because CF is caused by many mutations each modulator therapy works for different CF mutations.
The different mutations are grouped into different classes depending on how the production of protein is affected. Different modulator therapies work for different classes of mutation.
Learn more about the different classes of mutations
Different CFTR modulator therapies work on either one ar a few of the different genetic mutations that cause CF. Each class of mutation, for example, a gating mutation or a protein production mutation, affects the production of the CFTR protein differently, which is why some new precision medications only work for certain people with CF depending on their mutation.
Some medications are known as a combination therapy as it uses more two or more compounds to help the production or function of the CFTR protein. Triple therapy uses three drugs.
Ivacaftor (Kalydeco) was the first modulator therapy to be approved by the FDA in 2012 and since then 3 other precision medicines have been approved and are in use internationally.
Kalydeco - Ivacaftor (funded in NZ for G551D mutation)
Orkambi - Lumacaftor and Ivacaftor
Symdeko - Tezacaftor and Ivacaftor
Trikafta - Elexacaftor, Tezacaftor and IvacaftorLearn more about CFTR modulator therapies
On 26 February 2020, after years of campaigning, it was announced that Vertex and PHARMAC had reached a deal to fund Kalydeco in New Zealand. After a positive final consultation, Kalydeco became available in New Zealand from 1 March 2020.
We're committed to advocating for more precision medications for New Zealanders with CF.Visit our advocacy hub
With many new treatments and medications for cystic fibrosis already in use or in development there’s every reason to be hopeful of an improved quality of life and longevity for people with cystic fibrosis.
Drug development pipeline