Eli Lilly and Company / Alnara Pharmaceuticals

Exocrine Pancreatic Insufficiency (Cystic Fibrosis)

Disease Overview

Exocrine pancreatic insufficiency (EPI) is the inability of the pancreas to secrete enough digestive enzymes (lipase, protease, amylase) to digest food normally, causing malabsorption, steatorrhea, and malnutrition. In the US it most commonly arises from cystic fibrosis (affecting ~85% of CF patients), chronic pancreatitis, and pancreatectomy. Standard of care is lifelong pancreatic enzyme replacement therapy (PERT) taken with every meal.

Competitive Landscape

By 2009-2010 the FDA had completed a long-running enforcement action requiring all PERTs to obtain formal NDA approval, leaving five porcine pancrelipase products on the market: Abbott's Creon (the dominant brand), Aptalis/Eurand's Zenpep, J&J's Pancreaze, Digestive Care's Pertzye, and Aptalis's Ultresa. All five are pig-pancreas-derived enteric-coated capsules with similar mechanism but different particle sizes and dosing. Liprotamase was strategically differentiated as the only non-porcine recombinant PERT — a microbial-fermentation cocktail of cross-lipase, cross-protease, and cross-amylase — which Lilly believed would address supply-chain risk (porcine sourcing was contaminating concerns), religious/dietary objections, and dosing convenience. Cystic Fibrosis Foundation co-funded development. The thesis collapsed in 2011 when FDA issued a Complete Response Letter requesting additional efficacy data; Lilly took a $122.6M impairment in 2012 and divested liprotamase to Anthera in 2014, where it became Sollpura and ultimately failed Phase 3 [https://www.genengnews.com/news/anthera-buys-liprotamase-from-lilly-plans-phase-iii-trial/]. The deal is a textbook 'platform bet that lost the regulatory race' case study.