Atlanta, Georgia – Treatment with Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) may help reverse pancreatic dysfunction in young children with cystic fibrosis (CF), challenging the long-held belief that this complication is irreversible.
New findings presented by Vertex Pharmaceuticals, the therapy’s developer, at the North American Cystic Fibrosis Conference (NACFC) showed improvements in pancreatic function among children ages 2 to 5 who received Alyftrek for approximately one year.
According to Vertex, the results suggest that early treatment with CFTR modulators may help restore pancreatic function and reduce the need for pancreatic enzyme replacement therapy (PERT).
How Cystic Fibrosis Affects the Pancreas
Cystic fibrosis is a genetic disease caused by mutations in the CFTR gene, which provides instructions for making a protein that regulates the movement of salt and water across cell membranes. When this protein does not function properly, mucus becomes thick and sticky, accumulating in organs and contributing to the disease’s symptoms.
The pancreas is particularly vulnerable to these effects. It produces digestive enzymes that help the body break down fats, proteins, and carbohydrates. In people with CF, thick mucus can obstruct the ducts that carry these enzymes to the intestines, preventing proper digestion and nutrient absorption.
This condition, known as exocrine pancreatic insufficiency (EPI), affects the vast majority of children with CF by their first birthday. Treatment typically involves PERT, which supplies digestive enzymes to help compensate for the pancreas’s reduced function.
Until recently, pancreatic damage associated with CF and the resulting EPI were generally considered irreversible.
Alyftrek and Its Potential to Restore Pancreatic Function
Alyftrek combines three CFTR modulators that help improve the function of the defective CFTR protein in people with eligible genetic mutations. By improving CFTR activity, the treatment can help regulate mucus production and address some of the underlying mechanisms of CF.
Developed by Vertex as a next-generation successor to Trikafta (elexacaftor/tezacaftor/ivacaftor), Alyftrek is approved in the United States for eligible patients ages 6 and older.
Vertex is also evaluating the treatment in younger children through an ongoing Phase 3 clinical trial (NCT05844449), which includes patients as young as 1 year old.
Study Findings Show Improvements in Pancreatic Function
The newly presented findings came from a substudy involving 48 children ages 2 to 5 who completed approximately one year of follow-up in the Phase 3 trial. Researchers specifically investigated whether Alyftrek could improve pancreatic function enough for children with existing EPI to stop taking PERT.
After approximately one year of treatment, the results showed:
- 48.6% of children had stool pancreatic enzyme levels above the threshold typically associated with EPI, indicating improved pancreatic enzyme production.
- 37.5% of children were able to discontinue PERT for an average period exceeding eight weeks.
Vertex said these findings suggest that Alyftrek may help reverse pancreatic insufficiency in some young children with CF.
Across the broader group of 66 children ages 2 to 5 included in the Phase 3 trial, 50% did not require PERT at the latest follow-up, according to the company.
Findings Could Change the Understanding of Early CF Treatment
Carmen Bozic, MD, Vertex’s executive vice president of global medicines development and medical affairs and chief medical officer, said the findings highlight the potential benefits of treating CF early in life.
Bozic noted that pancreatic failure and EPI have traditionally been viewed as irreversible consequences of CF. However, the results in young children treated with Alyftrek suggest that pancreatic dysfunction may be reversible in at least some patients.
The findings offer encouraging evidence that early intervention with CFTR modulators could have benefits beyond managing respiratory symptoms, potentially improving digestive function and reducing reliance on enzyme replacement therapy.
Further research and follow-up will be important to establish how long these improvements last and which children are most likely to benefit.
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