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 been an important one for his daughter or other children he knows with the disease. He says that he would leave it to her doctor to decide whether she should take liprotamase instead of the pig-derived supplement she now takes, but he believes it’s important to have Lilly’s drug available as an option.

The Cystic Fibrosis Foundation, a nonprofit group based in Bethesda, MD, has funded development of liprotamase, in part because of concern that sick pigs could contaminate or limit the supply of the existing supplements. Both Healey and Marshall have been fundraisers for the CF Foundation, asking their friends and neighbors to give donations to support the development of new treatments like liprotamase.

Liprotamase, which was studied in multiple late-stage clinical trials, isn’t supposed to to require as many capsules to be taken as the existing enzyme treatmentsl. (It’s also a potential treatment for multiple disorders that involve pancreatic enzyme insufficiency, not just cystic fibrosis.) It could mean taking one or two capsules rather than three to five or more at each meal. For instance, Healey says that her son takes about 25 capsules of the pig-derived enzymes per day, and requiring that many pills is a major burden considering the battery of other treatments he needs to manage his disease.

While his first concern is as the dad of an 11-year-old girl with cystic fibrosis, Marshall says he learned about liprotamase through his work as a corporate recruiter for Altus Pharmaceuticals, the original developer of the treatment, nearly a decade ago. Altus ran into financial trouble and gave up rights to the drug to the CF Foundation, which then gave Alnara those rights in March 2009. His Portsmouth, NH-based firm, Stratacuity, has also worked for Alnara.

In July, Indianapolis-based Lilly bought Alnara for and upfront payment of $188.7 million and agreed to pay up to $200 million more to the biotech’s shareholders depending on certain regulatory and commercial goals being met. Alnara’s key shareholders include Bessemer Venture Partners, Frazier Healthcare Ventures, Longwood Founders Fund, MPM Capital, and Third Rock Ventures. Alnara is still operating as a unit of Lilly in Cambridge.

Lilly might be forced to make a tough decision about whether it wants to invest in another clinical trial of liprotamase, depending on the FDA’s ruling on its application for approval. Last week the company issued a statement that it is “confident” in the clinical trial data that has been submitted to the agency for the drug’s approval.

Still, what is often lost in all the high-stakes financial interests of drug development is what it all means for patients. Healey and Marshall provide vivid pictures of what the future of this drug could mean for people like their kids.

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