Modern treatment of cystic fibrosis will be covered by VZP for children from the age of two

Kaftrio for the Treatment of Cystic Fibrosis will now be available to children as young as two. Until now, it has only been given to patients over six. The extension of the indication was approved at the end of last year by the European Medicines Agency (EMA). General Health Insurance Company of the Czech Republic (VZP ČR) is now, in agreement with the professional society, lowering the age limit for its clients in the Czech Republic. The extension of the indication criteria comes less than two years after VZP started to cover the medicine Kaftrio for children over six years of age after meeting the given conditions. VZP has been reimbursing the drug to patients over 12 years of age since June 2021.

"Lowering the age of onset of this drug has great potential to slow the progression of the disease in specific patients, giving them a chance to significantly improve their quality of life. Currently, 39 of our young clients aged between 2 and 5 years are struggling with this disease. Early treatment prevents progression resulting in repeated hospital admissions for respiratory tract infections that gradually cause irreversible lung damage, for which lung transplantation is then the only treatment option. These patients also suffer from impaired pancreatic function, which causes poor digestion and related disorders. It is this irreversible damage that VZP wants to prevent by starting treatment as early as possible in its paediatric patients." explains Jan Bodnár, Deputy Director of VZP for Health Care.

The drug Kaftrio is intended for patients who have at least one F508del mutation in the CFTR gene in their genotype and is administered in combination with the LP Kalydeco. It is this combination that gives pediatric patients the best chance of improving their health status.

Prof. MUDr. Pavel Dřevínek, Head of the Institute of Medical Microbiology at the 2nd Faculty of Medicine of the Charles University in Prague and Motol University Hospital, Head of the Cystic Fibrosis Centre at Motol University Hospital says: "I very much welcome the shift of the age limit towards our smallest patients. By starting treatment early in the first years of a child's life, we can prevent the development of serious complications that otherwise develop based on cystic fibrosis and which are irreversible."

The General Health Insurance Fund of the Czech Republic will cover the treatment for patients in this age group who meet the conditions set by the Cystic Fibrosis Working Group of the Czech Society for Cystic Fibrosis.

Cystic fibrosis is an inherited congenital disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Patients with this disease are treated at specialised centres located at the Motol Hospital, Brno University Hospital, Olomouc University Hospital, Hradec Kralove University Hospital and Pilsen University Hospital. In the Czech Republic, there are currently 77 patients with cystic fibrosis aged between 2 and 5 years.

Source: VZP press release