Cystic fibrosis: appeal to Aifa for life-saving drug already approved by Ema
Kaftrio is already available in Italy, but only for one mutation: without a new authorisation, one in four young patients remains without treatment today
by Francesca Farma*.
2' min read
2' min read
Cystic fibrosis is a degenerative genetic disease with no cure. The only lifesaver available today is the drug Kaftrio, which greatly slows down, if not stops, the degeneration of organs, giving years of life. Life expectancy today for those not taking Kaftrio is 40 years, lived very badly. Overall in Italy we are talking about 6,000 patients.
Kaftrio is already available in Italy, for only one mutation, delF508, which is present in about 75% of patients
Cystic fibrosis, however, can be caused by more than 2000 mutations and therefore 25% of patients remain untreated to date.
The April decision of the European Medicines Agency
.In April 2025, the EMA authorised enlargement to include a large number of mutations (all but class 1), thus giving access to the life-saving drug even to those patients who until then could not take it.
Unfortunately, the timescales in Italy are always very long and to date our young people (in this case we are talking mostly about young adults), are not yet entitled to the treatment, whereas in most European countries it is already available.
