A new combination of cystic fibrosis drugs has been licensed for children two years and older.
A phase 3 study conducted in particular in Chicago has established that a combination of drugs against cystic fibrosis, namely “lumacaftor” and “ivacaftor”, is safe and effective in young children. The disease must, in this specific case, be caused by the mutation of the “CFTR” gene, which triggers the most common form of cystic fibrosis.
Authorization of this treatment for children “2 years and over”
These data, published in the Lancet Respiratory Medicine, served as a basis for the American health authorities, who authorized this treatment for sick children “2 years and older”. Previously, this drug combination was only given to patients “6 years and older”.
“With earlier treatments that target the genetic cause of cystic fibrosis, we hope to halt disease progression and significantly improve long-term outcomes,” says study author and professor of pediatrics Susanna A. McColley ( Northwestern University Feinberg School of Medicine). “In this study, we also observed an improvement in pancreatic function, suggesting that we may be able to repair the damage caused to the pancreas by the disease,” she adds.
Improve children’s growth
Cystic fibrosis is a progressive genetic disease that damages several organs, including the lungs and the pancreas. Patients survive on average only 47 years. It is caused by mutations within the CFTR gene. In the lungs, this creates a buildup of thick, sticky mucus that can lead to chronic lung infections and serious lung disease. Damage to the pancreas occurs even before birth, impairing the absorption of nutrients and therefore the growth of affected children.
“Our study also showed that the plumacaftor/ivacaftor treatment can improve the growth of children with cystic fibrosis”, continues Susanna A. McColley. “We observed a significant increase in weight, height and body mass index in six months of treatment” in treated patients, she concludes.
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