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

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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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