RARE Daily

FDA Creates New Advisory Committee of Genetic Metabolic Disease

December 12, 2023

Rare Daily Staff

The U.S. Food and Drug Administration said it is creating a new advisory committee related to potential treatments for genetic metabolic diseases.

Genetic metabolic diseases are conditions that disrupt an individual’s metabolism, which is the chemical process responsible for converting food into energy and eliminating toxins from the body. These diseases develop when a genetic flaw causes a protein or enzyme to be absent or faulty, interfering with certain vital metabolic functions. There are hundreds of different genetic metabolic diseases, most of which are rare and carry significant morbidity and can be life-limiting.

Advisory committees allow the FDA to receive input from clinicians, industry experts, academia, patients, caregivers and other external stakeholders when evaluating the potential benefits and risks of a new therapy. The advisory committee has been established to advise the agency on products used for the diagnosis, prevention or treatment of genetic metabolic diseases under the purview of the Division of Rare Diseases and Medical Genetics.

The group will be comprised of experts in the areas of metabolic genetics, management of inborn errors of metabolism, small population trial design, translational science, pediatrics, epidemiology, or statistics and related specialties. The committee will consist of nine voting members, including the committee chairperson. The committee will include a consumer representative and an industry representative.

“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 FDA’s 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.”

Photo: Janet Maynard, director of the FDA’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine


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