FDA's New Advisory Committee For Genetic Metabolic Diseases

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

The new Genetic Metabolic Diseases Advisory Committee would advise the agency on medical products used for the diagnosis, prevention or treatment of hundreds of different genetic metabolic diseases. Most of these diseases are rare and carry significant morbidity and can be life-limiting.

The new committee will consist of nine voting members, including the committee chairperson. It will include a consumer representative and an industry representative. Members will be invited to serve for overlapping terms of up to four years.

According to the agency, 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.

When called upon, the new committee will provide the FDA independent, knowledgeable advice and recommendations on technical, scientific and policy issues around medical products for such diseases.

For their advice and recommendations, the members will evaluate evidence on key issues about the applications brought before the advisory committee.

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

In October, the FDA had formed a new Digital Health Advisory Committee to help it explore the complex, scientific and technical issues related to digital health technologies or DHTs.

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