Cystic fibrosis: free access to new drugs extended to all patients
Negotiations concluded with an 'important discount' from the manufacturer. Aifa president Nisticò: our priority is to give citizens essential medicines
Key points
The long-awaited news comes from the Italian Medicines Agency for all cystic fibrosis patients (not only those with the DeltaF508 mutation) who will be able to benefit free of charge from the drug Kaftrio in combination with Kalydeco for all new indications authorised by the European Medicines Agency.
"This result," Aifa explains, "is the result of close negotiations conducted by the Scientific and Economic Commission with the pharmaceutical company that owns the medicine, Vertex Pharmaceuticals, which ended with an important (confidential) discount obtained by the Agency on the price proposed by the company at the start of negotiations in July 2025.
Approximately 5 thousand patients throughout Italy
The drug was already reimbursed in Italy for patients from 2 years of age with the most common mutations, which affect about 80% of patients (over 3,000 in Italy, according to estimates). With the extension to the rarest mutations, the number of patients who will have access to the treatment paid for by the National Health Service will also include almost all patients not yet included in the reimbursement scheme (about 1,600), who until now had no valid treatment alternatives.
'There was a lot of expectation on the part of patients,' said Aifa president, Robert Nisticò, 'and we are pleased that the Agency's commitment to discussions with the company has allowed the negotiation process to be successfully concluded, as it is our priority to make essential medicines available to citizens. Clearly it is Aifa's responsibility to ensure that the costs of these new therapies are sustainable for the National Health Service. This is why it is essential to assess innovation according to its real added value for patients and with a view to investing in the healthcare system as a whole'.
The disease is now a little less scary
"The more than two years of waiting since the application was made to EMA,' says Francesca Farma of the Cystic Fibrosis Research Foundation, 'have been erased by this latest approval step. The outlook and quality of life of 1,600 Italian patients has now changed, and this disease, to which our young people have to devote hours of their time and sacrifice so much, is finally a little less scary, and from being fatal, it is likely to become chronic'. Farma also emphasises the importance of 'not forgetting the patients who, because of the type of mutations they have, are still without a cure. Research has never stopped and goes on to find a definitive cure for them, as for all patients'.
