The objective of our study was to determine whether infants with cystic fibrosis who developed exocrine pancreatic insufficiency in early infancy would tolerate long-term treatment with ZENPEP (pancrelipase) delayed-release capsules, containing 3000 US Pharmacopeia units of lipase/capsule, and demonstrate consistent long-term growth. The most common treatment-emergent adverse events were diarrhea, vomiting, and constipation (mild or moderate). At study completion, median weight-for-age percentiles increased from 22nd to 49th, median length-for-age percentiles increased from 36.5th to 42nd, and median weight-for-length percentiles increased from 41.5th to 55.5th. Long-term treatment (up to 12 months) of infants with exocrine pancreatic insufficiency owing to cystic fibrosis with ZENPEP was well tolerated and associated with improved growth parameters. This is the first long-term study of pancreatic enzyme replacement therapy conducted in this patient population.

Pancrelipase is a porcine pancreatic extract which contains the digestive enzymes lipases, proteases and amylases. In patients with pancreatic exocrine insufficiency (PEI) from conditions such as chronic pancreatitis, pancreatectomy and cystic fibrosis, pancrelipase can be used as pancreatic enzyme replacement therapy (PERT). Pancrelipase can reverse steatorrhea, prevent weight loss, control pain and correct other nutritional deficiencies resulting from exocrine insufficiency. Various forms of pancreatic enzymes were being marketed as over-the-counter medications prior to the recent FDA declaration that all pancreatic enzyme products had to obtain approval as new drugs before marketing. On the basis of evidence from recent randomized controlled trials, three pancrelipase formulations (Creon®, Zenpep® and Pancreaze®) have been approved by the FDA as effective treatments for PEI. Although several tests exist for the detection of PEI, early diagnosis still remains a challenge. Individualization of the timing of treatment initiation and dosage requirements is needed to achieve optimal effectiveness. When used at recommended doses, pancrelipase is a safe medication. Appropriate use of pancrelipase in patients with pancreatic exocrine insufficiency can achieve symptomatic relief, prevent morbidity/mortality and also improve quality of life.