Patients’ Parents Upset By FDA Panel’s Rejection of Lilly/Alnara’s Cystic Fibrosis Drug

1/19/11

Sometimes it takes a parent’s perspective on the regulatory process for drugs in this country to understand what is really at stake for patients.

Last week an FDA advisory panel shot down Eli Lilly’s (NYSE:LLY) enzyme therapy for cystic fibrosis that has deep roots in the Boston area. And two parents of children with cystic fibrosis, a genetic disease that typically requires people to take such treatments, say they feel like the regulators got this one wrong.

“I appreciate that they are trying very hard not to do anything bad, but the measures that they were asking us to use for this particular product were meaningless,” says Francine Healey, a Needham, MA, mother of two children with cystic fibrosis.

Cystic fibrosis, which is known for causing a buildup of thick mucus in the lungs that makes it hard to breathe, also affects people’s digestion and their ability to absorb sufficient nutrients to maintain their weight. About 30,000 patients in the U.S. have the disease, and they have a median life expectancy of about 37 years. Liprotamase—the Lilly drug that it picked up through its buyout of Cambridge, MA-based Alnara Pharmaceuticals for as much as $380 million last summer—-is intended to provide certain pancreatic enzymes that patients with the disease and other conditions need to gain nutrition from foods. Yet the FDA panel voted 9-3 last week that there wasn’t enough evidence that the drug was effective for CF patients.

The FDA is expected to say whether it will approve liprotamase by mid-April. The agency isn’t required to follow the advice of its advisory panels, but it often does.

Healey, who testified in support of the drug at the panel’s meeting last week, has since written to FDA officials to let them know she disagreed with the outcome of the hearing. Her 19-year-old son, Mike, and her 16-year-old daughter, Amanda, now take digestion supplements made with enzymes from pigs. Liprotamase is made in a microbial process and would be the first approved treatment of its kind not to use the pig enzymes.

The FDA panel voted 11-1 in favor of recommending that additional studies of the effectiveness of liprotamase be done before it is approved. The agency’s staff also noted in its analysis issued last week that liprotamase scored lower in a measure of fat absorption than the existing supplements made with pig enzymes.

Patrick Marshall, who testified at the panel as a parent with a daughter who has cystic fibrosis, says that the measure of fat absorption has never … Next Page »

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