FDA Panel Rejects Lilly Pancreatic-Enzyme Replacement

WASHINGTON – A Food and Drug Administration panel rejected a new type of pancreatic-enzyme replacement product developed by an Eli Lilly & Co. (LLY) unit on concerns the drug isn't effective.

The panel said clinical data submitted in support of the drug, liprotamase, didn't "constitute substantial evidence of the efficacy," or effectiveness, in a 9-to-3 vote. The panel also said additional studies of liprotamase should be conducted prior to approval and potentially compared to current pancreatic-enzyme replacement products on the market.

The panel also said, in a 7-to-4 vote, that the benefits of liprotamase didn't outweigh the potential risks, which amounts to a recommendation that the FDA not approve the product. One person abstained on that vote.

Liprotamase, which has a proposed brand name of Sollpura, was reviewed Wednesday by outside medical experts on the FDA's gastrointestinal drugs advisory committee.

The product was developed by Alnara Pharmaceuticals to treat exocrine pancreatic insufficiency in children and adults caused by cystic fibrosis and other conditions. EPI is a lack of digestive enzymes made by the pancreas that results in the inability to properly digest food and absorb nutrients. Lilly bought Alnara last year.

In a statement, Lilly said it remained confident in liprotamase's clinical data. The FDA is expected to make a decision in April.

Unlike other pancreatic-enzyme replacement products, such as Abbott Laboratories' (ABT) Creon, liprotamase was developed without using enzymes taken from the pancreas of the pig.

The FDA has said there's a very small risk that viruses could be transmitted to patients with porcine-derived enzymes. Liprotamase is also designed to be given with just one pill per meal or snack unlike other drugs which can require multiple pills per meal.

Some parents of children with cystic fibrosis who spoke during the FDA panel meeting said up to 25 pancreatic-enzyme pills are needed each day and urged the panel to vote to approve liprotamase.

But the FDA questioned liprotamase's effectiveness. The agency said the main clinical study endpoint used to evaluate pancreatic-enzyme replacement products is a change in so-called coefficient of fat absorption, a measure of fat absorption.

"FDA reviewers have questioned whether the change in fat absorption observed with liprotamase is comparable to that observed with the porcine-derived [products]," the agency said in a summary document prepared for the meeting. If enough fat isn't absorbed from meals, it can results in weight loss and impaired growth in children.

In one of the main studies of liprotamase, the change in fat absorption was 11 percent compared to those receiving a placebo. The FDA said the change in the fat-absorption with other pancreatic enzyme products ranged from 26 percent to 41 percent.

Officials from Alnara cautioned against comparing studies of liprotamase to studies of other products partly because they had different designs. The 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.

Cystic fibrosis affects about 30,000 children and adults in the U.S. About 90 percent of cystic fibrosis patients receive pancreatic enzyme replacement therapy.