Innovative drug for cystic fibrosis extended to children aged 6-11 years

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Cystic fibrosis is a rare genetic disease that affects around 400 people in Portugal.

Infarmed has extended the use of the medicines Kaftrio and Kalydeco for the treatment of cystic fibrosis in the National Health Service to children aged between 6 and 11, the Medicines and Health Products Authority said.

According to Infarmed, public funding has been approved for the use, in combination, of both drugs in cases of children between 6 and 11 years old “who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene”.

The Medicines Authority also says that Kaftrio should only be prescribed by healthcare professionals with experience in treating cystic fibrosis and adds that if the patient’s genotype is unknown, “an accurate and validated genotyping method should be performed to confirm the presence of at least one F508del mutation”.

Cystic fibrosis is a rare genetic disease that affects around 400 people in Portugal. It causes deterioration of lung function, bronchopulmonary infections and malnutrition, which eventually lead to the patient’s death.

In March 2021, following the story of the young Constança Bradell, who exposed on social networks her fight against the disease and complained about the delay in the approval of Kaftrio in Portugal, Infarmed clarified that access to that innovative medicine was already possible since November 2020 through a Special Use Authorization (AUE) submitted by a hospital of the National Health Service.

The young woman eventually died in July 2021, aged 24.

Later, in December 2021, dozens of cystic fibrosis patients reiterated their call to Infarmed for faster access to this medicine – which at the time had already been approved by the European Medicines Agency and the European Commission.

Already in March 2022, the Medicines and Health Products Authority extended the use to a larger number of patients and, at the time, estimated an increase from 140 to around 215 of patients eligible for this therapy.

Until now, the use of the drug was authorized only in people aged 12 years and over.

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