Proposed Drug to Treat Certain Cystic Fibrosis Patients Should Not Be Approved; Trials Would Be Unethical

Jan. 12, 2011

Proposed Drug to Treat Certain Cystic Fibrosis Patients Should Not Be Approved; Trials Would Be Unethical

Dr. Michael Carome, Deputy Director of Public Citizen’s Health Research Group, Testifies Today Before FDA Committee

WASHINGTON, D.C. – Liprotamase, a new drug proposed to treat patients with cystic fibrosis who have problems digesting food, should not be approved by the Food and Drug Administration (FDA), and further proposed trials to determine the effectiveness of liprotamase would be unethical, Public Citizen told an FDA advisory committee today.

Data from initial studies show that liprotamase, a drug that replaces pancreatic enzymes for digesting food that are deficient in children and adults with cystic fibrosis and other diseases, is less effective than pancreatic enzyme products already approved by the FDA, Dr. Michael Carome, deputy director of Public Citizen’s Health Research Group, said. In addition, liprotamase, manufactured by Alnara Pharmaceuticals, a wholly owned subsidiary of Eli Lilly and Company, raises safety concerns related to potential inadequate growth, malnutrition, liver toxicity and intestinal disorders.

The FDA convened its advisory committee to address the question of whether the agency should approve liprotamase given the drug’s potential risks and decreased benefits.

Parents who are informed about the risks likely would not enroll their children in future randomized studies comparing the drug to older, more effective and safer FDA-approved products, Carome told the Gastrointestinal Drugs Advisory Committee. Further, such a randomized study would not satisfy the criteria for approval under FDA regulations concerning additional safeguards needed for children in clinical investigations.

“There are better, safer and more effective drugs other than liprotamase that are available for treating patients with cystic fibrosis,” Dr. Carome said.
 
Carome’s presentation can be found here,

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Public Citizen is a national, nonprofit consumer advocacy organization based in Washington, D.C. For more information, please visit www.citizen.org.