We were thrilled to share some very positive news last Friday (28th February) and we know that many in the CF community were excited to hear the news!   

After a long wait and a lot of effort from the CF community, this is a moment of hope that we must celebrate.  

The EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for a label extension of ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor, for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one non-class I mutation.  

What happens next? Watch the video where Audrey Chansard, a board member of CF Europe and a well-known patient advocate in the community, explains which steps will follow the EMA decision and when patients might access the treatment.

What changed?  

The EMA took an historical decision, based on a variety of data, including real-life studies. This approach could truly change the lives of many people with ultra rare diseases where, because of the small numbers of patients, clinical trials can’t be conducted. 

 

What happens next?  

A CHMP positive opinion is not a Marketing Authorisation decision, which means that the label extension is not yet licensed in the European Union. Marketing Authorisation decisions are taken by the European Commission separately, following CHMP opinion. This should happen within 67 days of receipt of the CHMP opinion. 

 

When will people with rare mutations have access to the treatment? 

After the decision of the Marketing Authorisation of the European Commission each country needs to decide on pricing and reimbursement again. It is difficult to predict timelines for that and it will differ from country to country. 

We remain available to respond to any questions from the CF community. Also, if patients have any questions, they can speak with their healthcare providers. 

Thank you for your continued support and dedication. 

 

Does this mean that Kaftrio will definitely work for me if I have at least 1 non-class 1 mutation? 

Unfortunately, it’s not that simple. The CHMP has deemed Kaftrio safe for the use in people with CF from the age of two who do not have an F508del mutation, excluding people with two stop mutations. However, we know that the medicine will most likely not work for everyone in this group. Data from a real-life study in France, that helped to come to this decision, show that over 50% of people in this group responds to Kaftrio, and that it is very much dependent on the mutations you have. Therefore, it is very important that start-up of the modulator therapy is done under expert guidance of the CF team who will evaluate whether you benefit from Kaftrio or not.