Safety and Efficacy of a Novel Microbial Lipase in Patients with Exocrine Pancreatic Insufficiency due to Cystic Fibrosis: A Randomized Controlled Clinical Trial

J Pediatr. 2016 Sep:176:156-161.e1. doi: 10.1016/j.jpeds.2016.05.049. Epub 2016 Jun 11.


Objective: To evaluate the safety and efficacy of a novel microbial lipase (NM-BL) in a liquid formulation for the treatment of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF) in a phase IIa proof-of-concept study.

Study design: We conducted a double-blind, randomized, placebo controlled crossover study in patients with cystic fibrosis and exocrine pancreatic insufficiency. Adolescent and adult patients with CF were randomized to receive NM-BL or placebo for 1 week as replacement for their usual pancreatic enzyme formulation. They were subsequently crossed-over to the alternate study treatment. The coefficient of fat absorption was evaluated as the primary endpoint. Symptoms and adverse events were evaluated as secondary endpoints.

Results: A total of 35 patients were randomized into the study and 22 patients completed both treatment periods. During treatment with NM-BL, the coefficient of fat absorption was significantly greater (72.7%) compared with placebo (53.8%) with a difference between groups of 18.8% (P < .001). Subjective assessment of stool fat and stool consistency also improved under treatment with NM-BL. Adverse events were mostly gastrointestinal in nature and were more common in the group receiving NM-BL.

Conclusions: Currently available pancreatic enzyme products are limited because of the lack of liquid formulations and being largely porcine based. The novel microbial lipase NM-BL was safe and effective in this short term trial. The trial provided clinical proof-of-concept for this novel microbial lipase as a treatment for EPI in CF. A larger phase 2 dose ranging trial is warranted.

Trial registration: NCT01710644.

Keywords: clinical trial; cystic fibrosis; exocrine pancreatic insufficiency; pancreatic enzyme-replacement therapy.

Publication types

  • Clinical Trial, Phase II
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Child
  • Cross-Over Studies
  • Cystic Fibrosis / complications
  • Double-Blind Method
  • Exocrine Pancreatic Insufficiency / drug therapy*
  • Exocrine Pancreatic Insufficiency / etiology
  • Female
  • Humans
  • Lipase / adverse effects
  • Lipase / therapeutic use*
  • Male
  • Treatment Outcome
  • Young Adult


  • solysime
  • Lipase

Associated data