CHMP positive opinion for Triple Combination of CFTR modulators

We are pleased to inform you that the CHMP, the European Committee for Medicinal Products for Human Use, has given a positive opinion for the use of the triple combination of CFTR modulators Kaftrio® (Vertex Pharmaceuticals) for patients above 12 years of age, homozygous for F508del and heterozygous with F508del and a broad range of minimal function mutations.

The positive opinion of the CHMP quickly followed the letter CF Europe wrote to the European Medicines Agency (EMA) in May, requesting a swift decision on market authorisation for the triple combination of CFTR modulators, especially given the challenges people with CF face during the COVID-19 pandemic. Although we do not know to what extent our letter helped push things forward, we are pleased to see that the CHMP included a broad range of mutations in its opinion, including (rare) mutations that were not tested during the clinical trials. We hope that this may create a precedent for granting market authorization for innovative drugs for people with CF with rare genotypes that cannot all be tested during a clinical trial, maybe even in other disease areas as well.

Importantly, whilst this is a positive step, the dossier will now need to be reviewed by the European Commission before marketing authorisation is granted. This process usually takes about 2 months. CF Europe will continue to follow up on this and inform you about any decision.

You can find the CHMP positive opinion for Kaftrio® here.



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