FDA panel rejects new pancreatic enzyme replacement drug

By Dr. Ananya Mandal, MDJan 12 2011

Eli Lilly & Co did not win the support of the U.S. advisory panel on Wednesday for its one-of-its kind drug not made from pig enzymes to help certain pancreatic patients digest food. A Food and Drug Administration panel of outside experts in a 7-4 vote said they were not convinced the experimental medicine, known chemically as liprotamase, worked well enough for patients to absorb enough needed nutrients. One member of the panel abstained.

The panel was concerned that liprotamase does not appear to work better than currently approved treatments derived from pigs, or “porcine derived.” Panelist Ronald Fogel, a Michigan-based physician said, “We know it’s better than placebo, but we don't know if it's as good as the porcine products.” In one of the main studies of liprotamase there was a 11% change in fat absorption compared to those receiving a placebo. The FDA said the change in the fat-absorption with other pancreatic enzyme products ranged from 26% to 41%.

At present Lilly’s Alnara Pharmaceuticals Inc subsidiary is seeking FDA approval to market the drug to patients with pancreatic insufficiency due to various causes, such as cystic fibrosis, in order to help them digest food. The drug is derived from microbial enzymes. In a statement after the meeting, Lilly said it remained confident in the clinical trial data submitted to the FDA. Hereafter the FDA will consider the panel’s opinion before granting approval or rejecting the drug.

The panelists called for more data, especially trials comparing it to currently-approved products as well as for more data on children. Patients with pancreatic insufficiency from cystic fibrosis or other conditions currently must take dozens of pills a day when they eat food to help them properly digest and absorb nutrients. The Alnara drug is claiming to reduce the pill burden for patients so that just 5 to 10 pills were needed on average. Several patient groups also urged the panel to give them another option. Jesse Joad of the University of California at Davis was skeptical saying a smaller pill load may be well but a less effective drug may hinder growth especially in younger patients. Panel chairman and University of Maryland medical professor Jean-Pierre Raufman, echoing other panelists, said, “I was not convinced that the data showed meaningful efficacy for this agent although it's obviously greatly needed.”

Officials from Alnara warned against comparing studies of liprotamase to studies of other products partly because they had different designs. The results from the studies showed that patients who stayed on liprotamase for up to a year showed little change in a measure of body mass index, suggesting the product was effective at meeting nutritional goals.