Infarmed has extended the use of Kaftrio and Kalydeco drugs for the treatment of cystic fibrosis to children between 6 and 11 years old in the National Health Service, informed the Medicines and Health Products Authority.
According to Infarmed, public funding was 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 gene (CFTR) ”.
The Medicines Authority also says that Kaftrio should only be prescribed by health professionals with experience in the treatment of cystic fibrosis and adds that, if the patient's genotype is unknown, "an accurate and validated genotyping method should be carried out 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 death of the patient.
In March 2021, following the story of the young Constança Bradell, who exposed her fight against the disease on social media and complained about the delay in approving 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 ended up dying in July 2021, aged 24.
Later, in December 2021, dozens of patients with cystic fibrosis reiterated their appeal 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.
In March 2022, the Medicines and Health Products Authority extended its use to a larger number of patients and, at the time, estimated an increase from 140 to around 215 patients eligible for this therapy.
Until now, the use of the drug was authorized only in people aged 12 years or older.
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