Newly approved medicines for Cystic Fibrosis

Cystic fibrosis is a rare, life-threatening genetic disorder that affects more than 70,000 people worldwide. It is caused by a mutation of the CFTR gene, which regulates salt and water transport in the body. The CFTR mutation allows too much salt and water into cells. This results in a build-up of thick, sticky mucus in the body's tubes and passageways. These blockages damage the lungs, digestive system and other organs. These symptoms start in early childhood and include persistent cough, recurring chest and lung infections and poor weight gain.

There are several treatments today that help with the management of Cystic Fibrosis. The  newest approved medicine for Cystic Fibrosis by the European Medical Agency (EMA) is Kaftrio (elexacaftor/tezacaftor/ivacaftor). 

The approval of Kaftrio (elexacaftor/tezacaftor/ivacaftor) by the EMA in August of 2020 comes after the approval of Trikafta (elexacaftor/tezacaftor/ivacaftor; ivacaftor) in October 2019. 

Here is an overview of each medicine to help guide you and your physician when making a treatment decision for Cystic Fibrosis: 

Trikafta (elexacaftor/tezacaftor/ivacaftor; ivacaftor)

On October 21, 2019 Trikafta (elexacaftor/tezacaftor/ivacaftor; ivacaftor) was approved by the Food and Drug Administration (FDA), USA, for the treatment of people aged 12 plus with certain forms of cystic fibrosis (CF).

Trikafta (elexacaftor/tezacaftor/ivacaftor; ivacaftor) is a combination of ivacaftor, tezacaftor, and elexacaftor indicated for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It is available in co-packaged fixed-dose combination tablets containing elexacaftor 100 mg, tezacaftor 50 mg and ivacaftor 75 mg, and tablets containing ivacaftor 150 mg. 

Kaftrio (elexacaftor/tezacaftor/ivacaftor)

On August 21, 2020 Kaftrio (elexacaftor/tezacaftor/ivacaftor) received an EMA marketing authorisation valid throughout the EU for use in combination with Kalydeco (ivacaftor) to treat people aged 12 plus with certain forms of cystic fibrosis (CF).

Kaftrio (elexacaftor/tezacaftor/ivacaftor) is indicated in a combination regimen with ivacaftor 150 mg tablets (not included in package) for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It is available as fixed-dose combination tablets containing elexacaftor 100 mg, tezacaftor 50 mg and ivacaftor 75 mg

You can download the complete medicines infographic here. 

If you or someone you know are looking to access Trikafta (elexacaftor/tezacaftor/ivacaftor; ivacaftor) or Kaftrio (elexacaftor/tezacaftor/ivacaftor) in combination with Kalydeco, we can help. 

Contact our Patient Support Team for more information.