EUR-1008 pancreatic enzyme replacement is safe and effective in patients with cystic fibrosis and pancreatic insufficiency.
BACKGROUND
EUR-1008 (Zenpep [pancrelipase]) is a new, enteric-coated, porcine-derived pancreatic enzyme product (PEP) developed for the treatment of cystic fibrosis (CF) patients with malabsorption associated with exocrine pancreatic insufficiency (EPI). Unlike currently marketed PEPs, EUR-1008 contains the label-claimed lipase content. Safety and efficacy were assessed in younger (<7 years) and older (> or =7 years) CF patients with EPI.


METHODS
Two multicenter studies were conducted: a randomized, double-blind, placebo-controlled, crossover trial in patients > or =7 years of age (N=34) and a supplemental, open-label study in children <7 years of age (N=19). Use of any medications altering gastric pH/motility was prohibited during the studies. Outcome measures in the randomized trial included changes in the coefficient of fat absorption (CFA), coefficient of nitrogen absorption (CNA), and signs/symptoms of malabsorption for EUR-1008 vs. placebo. Outcome measures in the supplemental study included safety and response (defined as no steatorrhea and no overt signs/symptoms of malabsorption) to EUR-1008 vs. previous enzyme treatment.


RESULTS
In the randomized trial, EUR-1008 treatment compared to placebo resulted in a significantly higher mean CFA (88.3% vs. 62.8%, respectively) and CNA (87.2% vs. 65.7%, respectively) (both p<0.001) and reduced the incidence of malabsorption signs and symptoms in 32 evaluable patients. In the supplemental study, 11 of 19 patients met the criteria for responder with EUR-1008 at the end of the study vs. 10 of 19 patients at screening (previous PEP), and improvements in clinical symptoms were reported with EUR-1008 treatment. EUR-1008 was safe and well tolerated, and no serious drug-related AEs were reported in either study.


CONCLUSIONS
EUR-1008 was safe, well tolerated, and effective in CF patients of all ages with EPI-associated malabsorption in two clinical trials. Treatment led to clinically and statistically significant improvements in CFA and CNA in the randomized study, and control of malabsorption and clinical symptoms in both studies.
Wooldridge
Jamie L
JL
Cincinnati Children's Hospital Medical Center, Division of Pulmonary Medicine, ML 2021, 3333 Burnet Ave., Cincinnati, OH 45229, USA. jamie.wooldridge@cchmc.org
Heubi
James E
JE
Amaro-Galvez
Rodolfo
R
Boas
Steven R
SR
Blake
Kathryn V
KV
Nasr
Samya Z
SZ
Chatfield
Barbara
B
McColley
Susanna A
SA
Woo
Marlyn S
MS
Hardy
Karen A
KA
Kravitz
Richard M
RM
Straforini
Cristina
C
Anelli
Marco
M
Lee
Candace
C
D000293:Adolescent; D002648:Child; D002784:Cholesterol / Q000097:blood; D018592:Cross-Over Studies; D003550:Cystic Fibrosis / Q000150:complications*; D010188:Exocrine Pancreatic Insufficiency / Q000188:drug therapy* / Q000209:etiology*; D005260:Female; D006801:Humans; D008286:Malabsorption Syndromes / Q000188:drug therapy / Q000209:etiology; D008297:Male; D020799:Pancrelipase / Q000008:administration & dosage* / Q000009:adverse effects; D013608:Tablets, Enteric-Coated; D016896:Treatment Outcome; D014815:Vitamins / Q000097:blood; D055815:Young Adult
D013608:Tablets, Enteric-Coated; D014815:Vitamins; D020799:Pancrelipase; D002784:Cholesterol