FDA Establishes Specialized Advisory Committee for Genetic Metabolic Diseases

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In a recent announcement, the U.S. Food and Drug Administration (FDA) revealed its plans to form a new advisory committee dedicated to addressing potential treatments for genetic metabolic diseases. These rare conditions disrupt an individual’s metabolism, a process crucial for converting food into energy and eliminating toxins from the body. A genetic flaw causing an absent or faulty protein or enzyme is the root cause of these diseases, interfering with essential metabolic functions.

The newly formed Genetic Metabolic Diseases Advisory Committee will serve as a knowledgeable source of independent advice to the FDA. It will provide critical insights and recommendations on technical, scientific, and policy matters concerning medical products developed for treating genetic metabolic diseases. The committee will consist of experts across various fields, including metabolic genetics, inborn errors of metabolism management, small population trial design, translational science, pediatrics, epidemiology or statistics, and other related specialties.

Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research (CDER), emphasized the importance of such advisory committees, stating, “Advisory committees allow the FDA to receive invaluable input from clinicians, industry experts, academia, patients, caregivers, and other external stakeholders when evaluating the potential benefits and risks of a new therapy. They are an essential part of the FDA’s work.”

The advisory committee falls under the Division of Rare Diseases and Medical Genetics, a division established in CDER’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine (ORPURM) in 2020. It will advise the agency on products used for diagnosing, preventing, or treating genetic metabolic diseases.

Janet Maynard, M.D., M.H.S., director of ORPURM, accentuated the unique challenges associated with drug development for these rare conditions. She expressed optimism that the new advisory committee would facilitate discussions on complex issues with specialized and diverse technical and scientific experts in the field of metabolic genetics.

The committee will comprise nine voting members, including the committee chairperson. All nominated scientific members must be technically qualified experts in their fields with experience in interpreting complex data. The committee will also include a consumer representative and an industry representative. Non-Federal members of this committee will serve either as special government employees or non-voting representatives, with terms lasting up to four years.

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