Eli Lilly & Co got its application for approval of its experimental medicine, liprotamase, rejected by FDA's Drugs Advisory Committee.
Liprotamase was claimed to be the first drug not made from pig enzymes that were routinely used in other drugs, to help patients with pancreatic disorders digest their food. But the panel of experts stated that there was not enough evidence to prove that liprotamase worked better than "porcine derived" drugs already in the market. Not being convinced, they wanted more information of trials comparing it to other products as well as data on the effect on children.
AdvertisementAlthough patients with pancreatic insufficiency may be attracted to taking just a few pills a day that liprotamase promises, compared to the dozen they had to take earlier to digest and absorb the nutrients from their food, they might not be benefited by a drug that has not been proven yet and that may work less effectively, hindering growth.
Panel chairman and University of Maryland medical professor Jean-Pierre Raufman, stated very emphatically, "I was not convinced that the data showed meaningful efficacy for this agent although it's obviously greatly needed."
It has caused a lot of distress, especially to parents with children suffering from pancreatic diseases to discover that the earlier drugs are derived from pigs and that they have no alternative now. Also, the company stands to lose heavily with its application being rejected.
Nevertheless, the panel has decided that more studies on the efficacy of the liprotamase should be conducted before approval, including studies that directly compared it with porcine products and measured effects of treatment on height, weight, and body mass index over time, as well as the effect on symptoms such as flatulence, steatorrhea, and stool frequency, and on quality of life measures.