Liprotamase powder is a non-porcine, soluble and stable mixture of three digestive enzymes including lipase, protease, and amylase. The purpose of the present study is to provide additional efficacy and safety data compared to approved, porcine-derived, enterically-coated and encapsulated pancreatic enzyme replacement therapy. The primary efficacy endpoint of the study will be comparative efficacy measured as the change in the coefficient of fat absorption (CFA) in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI).

Liprotamase is stable in stomach and digestive fluids allowing administration in a variety of convenient formulations and with a number of foods without enteric coating.

Porcine derived enzymes are used for pancreatic enzyme replacement therapy in patients with cystic fibrosis (CF). Liprotamase is a biotechnology-derived enzyme replacement without enteric coating. This is an open-label, assessor blind, parallel group, multicenter, international trial to evaluate the noninferiority of liprotamase and pancrelipase in CF patients aged ≥7 years with pancreatic insufficiency. Subjects were randomized to liprotamase or pancrelipase, dose-matched to pre-study lipase doses. The lower bound of the 95% confidence interval (CI) for noninferiority was -15% for treatment difference in change from baseline coefficient of fat absorption (CFA).

Inclusion Criteria:

• Diagnosis of Cystic Fibrosis based on presentation, genotype and/or sweat chloride
• Fecal elastase <100 mcg/g stool
• Minimum Coefficient of Fat (CFA) at screening while on stable PERT therapy
• Good nutritional status

Exclusion Criteria:

• History or diagnosis of fibrosing colonopathy
• Distal intestinal obstruction syndrome in 6 months prior to screening
• Receiving enteral tube feedings
• Chronic diarrheal illness unrelated to pancreatic insufficiency
• Liver abnormalities, or liver or lung transplant, or significant bowel resection
• Forced expiratory volume in 1 second (FEV1) <30%