FDA Announces Advisory Committee for Genetic Metabolic Disease Treatments

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The new advisory committee will provide the agency with technical and scientific advice and recommendations on potential treatments for genetic metabolic diseases.

FDA announced on Dec. 12, 2023 that the agency is creating the Genetic Metabolic Diseases Advisory Committee, which will provide independent advice and recommendations on technical, scientific, and policy issues regarding treatments for genetic metabolic diseases. The committee will advise FDA on products used to diagnose, prevent, or treat metabolic diseases under the purview of the Division of Rare Diseases and Medical Genetics.

Committee members will include experts in metabolic genetics, management of inborn errors of metabolism, small population trial design, translational science, pediatrics, epidemiology or statistics, and related specialties. There will be nine voting members, and there will be a consumer representative and an industry representative. Members will serve overlapping terms of up to four years.


“Advisory committees allow [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 [FDA’s] work,” said Patrizia Cavazzoni, director of FDA’s Center for Drug Evaluation and Research (CDER). “These expert panels support the agency by helping ensure [FDA’s] decisions on the safety and efficacy of drug products are informed and in line with our priorities and goals.”

Genetic metabolic diseases impact a person’s metabolism and develop when a genetic flaw causes a protein or enzyme to be faulty or non-existent. Most of these diseases are rare and can be life limiting, according to FDA.

“Genetic metabolic diseases include very rare diseases that individually affect a limited number of patients. Drug development for these conditions has unique and complex challenges, therefore few treatments are available to patients,” said Janet Maynard, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine. “This new advisory committee will provide a forum for discussion of complicated issues with specialized and diverse technical and scientific experts in the field of metabolic genetics.”

Source: FDA