As advocates for the Cystic Fibrosis (CF) community, CF Europe is committed to facilitating the approval and equitable access to life-enhancing and life-saving therapies for all people living with CF in Europe.

Today, we urge the European Medicines Agency (EMA) to extend the current license agreement of Kaftrio, which is proven to be safe and appropriate, to include people living with CF who were not eligible to participate in clinical trials due to their CFTR variants falling outside of the inclusion criteria. CF Europe argues for a non-conservative use of evidence, taking into account the amplitude of available evidence supporting the efficacy of Kaftrio in this group of over 3,000 people, including in vitro data and real-world data.

A recent French real-life study conducted by Professor Pierre-Régis Burgel, including over 500 people with CF, demonstrated that Kaftrio offers beneficial effects to more than 50% of patients whose CF is caused by variants other than F508del. In Europe, the marketing authorization for Kaftrio is currently intended only for patients carrying at least one F508del mutation. Following this study and lobbying from French CF patient associations and CF centers, all people with CF living in France are now able to access this treatment after a clinical assessment. This example shows the value in considering a broader range of data to demonstrate the efficacy of a treatment.

“If EMA accepts this type of evidence to extend Kaftrio towards people with CF who do not have F508del, it would not only be an important step for CF, but also a giant leap forward for all (ultra)rare diseases,” said Thierry Nouvel, President of CF Europe.

Such barriers to new treatments are not unique to CF but are also evident in other rare disease treatments. Given the myriad of rare disease treatments coming to market, we hope this situation will act as an example that will lead to broader approval of other treatments that may benefit patients. Without new methodologies, tens of thousands of people with (ultra-)rare diseases in Europe risk being left behind.

CF Europe will continue advocating for the interests of all people with CF to live their lives to the fullest potential. Those who could benefit should have the option to try this innovative treatment and, if beneficial, have sustained access to it. As advocates for the CF community, we offer our collaboration to all parties involved to work on a new, future-proof approach to the use of data. We call on them to come to a durable solution for all impacted people in Europe.

About CF Europe

CF Europe is the federation of national CF associations in Europe, dedicated to improving the lives of people living with CF through advocacy, research, and collaboration.

Media Contact:

Lidia Salvatori
Policy and Communications Manager
E: info@cf-europe.eu