Three Drugs Regimen Exhibit Safer Efficacy Against Cystic Fibrosis

Regimen of three drugs that target the genetic cause of most commonly affecting cystic fibrosis was found to be safe and effective in 6-11-year-olds.

Regimen of three drugs (elexacaftor/tezacaftor/ivacaftor) that targets the genetic cause of most commonly affecting cystic fibrosis was found to be safe and effective in 6-11-year-olds as per an international, open-label Phase 3 study at the Children’s Hospital of Chicago, published in the American Journal of Respiratory Care and Critical Care Medicine.

The three-drug cystic fibrosis treatment was approved by the FDA in October 2019 for people 12 years and older with at least one copy of F508del mutation. The mutation in the CFTR gene with one copy of F508del is estimated to represent almost 90 per cent of the cystic fibrosis population in the United States.

Cystic Fibrosis - Symptoms, Causes, Diagnosis, Treatment, Support Groups
Cystic fibrosis is a genetic disease involving the mucus and sweat glands and the medical world has still to find its cure

‘Regimen of three drugs (elexacaftor/tezacaftor/ivacaftor) that targets the genetic cause of most commonly affecting cystic fibrosis was found to be safe and effective in 6-11-year-olds. Significant improvements in lung function, respiratory symptoms and nutritional status was also observed from the treatment. ’

Cystic fibrosis is a progressive genetic disease that damages multiple organs, including the lungs and pancreas with an average life expectancy of 47 years. The mutations in the CFTR gene that lead to insufficient flow of salt and water in and out of cells is responsible for causing the disease.

This results in a build-up of thick, sticky mucus that leads to chronic lung infections and severe lung disease. The FDA has accepted the application to expand treatment indication to younger children, with a decision expected by June 2021, based on the positive results of this study.

“The most exciting aspect of our findings is that this population of children had normal lung function at the start of the study and still had a significant improvement. Coupled with what we saw in studies and practice with the older population, starting treatment earlier may avert serious long-term complications and change the trajectory of health for children with cystic fibrosis”, says Dr. McColley, Co-Global Principal Investigator on the study and senior author, who is the Scientific Director for Interdisciplinary Research Partnerships at Stanley Manne Children’s Research Institute at Lurie Children’s and Professor of Pediatrics at Northwestern University Feinberg School of Medicine.

Urine Test for Cystic Fibrosis Patients is Beneficial: Study
A urine test in patients with cystic fibrosis could be beneficial, according to a new study. The urine test will help analyze the cystic fibrosis transmembrane conductance regulator in patients.

Three Drug Regimen for Cystic Fibrosis

The research team carried a 24-week study with 66 children and confirmed the appropriateness of a dose. It was formulated to half of the adult daily dose of the three-drug treatment for children 6-11 years of age who weigh less than 30 kg and of the full adult dose for those weighing more.

Therapy for Cause of Cystic Fibrosis Safe and Effective
An international, open-label Phase 3 study, co-led by Susanna McColley, MD, from Ann & Robert H. Lurie Children's Hospital of Chicago, found that a regimen was safe and effective in 6-11-year-olds.

They observed that the regimen was well tolerated, and the safety profile was generally consistent with that observed in older patients, with cough, headache and fever as the most common adverse events.

Significant improvements in lung function, respiratory symptoms and nutritional status resulted from the treatment. Maintaining or improving nutritional status is associated with better lung function and increased survival in patients with cystic fibrosis.

Moreover substantial improvement in sweat chloride concentration, a direct measure of CFTR function, was also observed.

“In this study, we saw greater improvements in sweat chloride than those previously seen in adults and adolescents. This strong response to treatment may lead to better long-term clinical outcomes of cystic fibrosis. It’s important to note that people with cystic fibrosis who are demographically characterized as having a race other than white or ethnicity characterized as Hispanic are less likely to have an F508del mutation. This is important because as with other acute and chronic conditions, these populations have more severe disease and lower life expectancy. As drug development continues, we are focused on having a highly effective treatment or cure for everyone with cystic fibrosis”, says, Dr. McColley.

Source-Medindia