Infarmed Extends SNS Funding of Cystic Fibrosis Drugs to Patients Without the Common Mutation

People with cystic fibrosis in Portugal whose genes have so far ruled them out of the newest treatments can now be treated in National Health Service (SNS) hospitals. Infarmed (the National Authority for Medicines and Health Products) approved two funding decisions on 23 September and published them, with its assessment reports, on 1 October.

Both medicines, made by Vertex Pharmaceuticals, act on the faulty CFTR protein behind the disease rather than only on its symptoms.

What was approved

• Kaftrio with Kalydeco (elexacaftor, tezacaftor and ivacaftor, taken with extra ivacaftor) for patients aged 2 and over who do not carry the common F508del mutation, provided they have at least one mutation that is not Class I. Until now the SNS funded Kaftrio only for patients with at least one F508del copy.
• Alyftrek (deutivacaftor, tezacaftor and vanzacaftor), a newer once-daily tablet, for patients aged 6 and over with at least one mutation that is not Class I. The European Union authorised it on 30 June 2025.

Class I mutations stop the body making the protein at all, leaving these drugs nothing to work on, so patients with only Class I mutations remain outside both decisions.

Who stands to gain

Infarmed's assessment reports put the number of people living with cystic fibrosis in Portugal at about 413. Some 82 percent carry at least one F508del mutation and were already covered. The 17 to 18 percent without it are the group the Kaftrio extension targets. Many had only supportive care such as antibiotics. Infarmed estimates that about 5 percent of patients carry none of the mutations these medicines are listed for, so they still have no disease-modifying option. Neither report gives a count of newly eligible patients.

For the Kaftrio extension, Infarmed found a "major" added therapeutic benefit over supportive care. In the main trial of 307 patients aged 6 and over, exacerbations fell by 72 percent. The approved indication covers 152 mutations that responded in laboratory tests, compared with 18 in the trial. Infarmed accepted that for a rare disease, noting added uncertainty, including for children aged 2 to 5.

Alyftrek did not show added benefit over Kaftrio. It was funded because it came out cheaper in a cost comparison. Infarmed also counts 112 mutations in Alyftrek's label that Kaftrio's label does not cover.

How patients get it

Both medicines are restricted prescriptions for use in SNS hospitals, under contracts between Infarmed and the manufacturer. Cystic fibrosis medicines have long been fully funded and collected from hospital pharmacies under Despacho n.º 24/89.

A five-year build-up

Kaftrio first reached patients through special use authorisations in November 2020. The SNS approved it for patients aged 12 and over in July 2021, after the case of Constança Braddell, a 24-year-old patient who had campaigned for access and died that month. In March 2022 funding was extended to more mutations, raising the eligible group from about 140 to about 215 patients. Children aged 6 to 11 followed in July 2023 and children aged 2 to 5 in April 2025.

What remains open

The agreed prices are not published. Infarmed says only that it negotiated "more favourable conditions" for the SNS. The Alyftrek decision came about 15 months after EU authorisation, faster than the 650 days that a study reported in May as the usual wait between EU approval and an SNS funding decision; neither report says when Vertex applied. Both were assessed under Decreto-Lei n.º 97/2015, the funding regime replaced on 1 July 2026, because the applications predate it. Infarmed consulted the two patient associations, the Associação Portuguesa de Fibrose Quística and the Associação Nacional de Fibrose Quística, during the assessment; neither website carried a reaction by Wednesday evening.