Treatment for CF with Kaftrio

Kaftrio (also known as Kalydeco) is a groundbreaking, disease-correcting treatment for cystic fibrosis. Studies have shown that it can improve lung function by up to 14 percentage points in people with CF who have at least one F508del mutation in the CFTR gene.

In Norway, Kaftrio/Kalydeco was officially approved for public funding on April 25, 2022, by the Norwegian Decisions Forum. On May 27, 2024, it was also approved for use in children aged 2-6 years. Starting July 1, 2024, Kaftrio will be available for children with cystic fibrosis who have at least one F508del mutation.

Information about the Disease

Kaftrio

Treatment

Rights, Assistance, and Support

Facts about cystic fibrosis

What is Kaftrio/Kalydeco?

Kaftrio (also known as Kalydeco) is a cutting-edge, disease-correcting treatment for cystic fibrosis.

It combines three active ingredients - elexacaftor, tezacaftor, and ivacaftor.

Together they work to target the CFTR protein responsible for cystic fibrosis. This treatment is known as a CFTR modulator.

Preparatomtale Kaftrio på NORSKFor more details on specific minimal function mutations studied, check the Kaftrio product information (Section 5.1, pages 11-12) on the Norwegian Medicines Agency website.
Comprehensive approval details can be found on the EMA website.

When you cannot use Kaftrio

For those who cannot use Kaftrio, it can be extra difficult in a community characterized by collective jubilation. As an NFCF member, Bernhard (30) describes it so well: "It can feel like being left at the bus stop when the bus has left." Click on the link below to read more about this important topic, and how NFCF, together with international partners, works to ensure future treatment options for more people with CF.

Being left at the bus stop - When you can't use Kaftrio

Who can receive Kaftrio/Kalydeco?

Kaftrio/Kalydeco is approved for use in people with cystic fibrosis who are 6 years or older and have either one F508del mutation and one minimal function mutation (heterozygotes) or two F508del mutations (homozygotes). Starting July 1, 2024, Kaftrio will also be available in Norway for children aged 2-6 years who have at least one F508del mutation.

Best treatment for those with one F508del and minimal function mutation

Kaftrio/Kalydeco is the most effective disease-correcting medication for individuals with one F508del mutation and one minimal function mutation. It represents a significant advancement in cystic fibrosis treatment. Minimal function mutations lead to CFTR proteins with very limited activity, but Kaftrio/Kalydeco helps increase the amount of active CFTR protein

Effectiveness of Kaftrio/Kalydeco

Studies have shown that Kaftrio/Kalydeco can improve lung function by up to 14 percentage points in FEV1 compared to a placebo in people with one F508del and one minimal function mutation. For those with two F508del mutations, it has been shown to increase lung function by 10 percentage points compared to Symkevi, a previous CFTR modulator.

Kaftrio/Kalydeco contributes to a healthier CF population with fewer chronic infections, fewer exacerbations, and better nutritional status. Common side effects include acne, headache, diarrhea, and nasal congestion. The medication is taken as tablets twice daily.

Ongoing monitoring by healthcare professionals is essential to manage both the effectiveness and any potential side effects of the treatment.

600 Days of Battle

The fight to get Kaftrio/Kalydeco approved in Norway was long and arduous, spanning over 600 days. The Norwegian Cystic Fibrosis Association was steadfast throughout the entire process, and we can now look back with pride at the results of our efforts. The approval in 2022 for patients aged 6 and older, along with the crucial extension in 2024 to include children aged 2-6 years, represents significant victories for the CF community.

These achievements reflect our dedication and perseverance in overcoming substantial challenges. Whether the treatment will be approved for other mutation combinations in the future remains uncertain. However, ongoing drug development and research provide hope for those who currently do not meet the criteria for Kaftrio/Kalydeco.

As a member of the Norwegian Cystic Fibrosis Association, you play a vital role in supporting our efforts. Your support amplifies our voice with policymakers and decision-makers, helping us achieve these important milestones for you and your family.