The FDA announced 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.
When called upon, the Genetic Metabolic Diseases Advisory Committee will provide the FDA independent, knowledgeable advice and recommendations on technical, scientific and policy issues around medical products for genetic metabolic diseases. Committee members will evaluate evidence on key issues about the applications brought before the advisory committee and offer their recommendations for FDA consideration. 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 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. This division, established in CDER's Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine (ORPURM) in 2020, oversees the development of these medical products for genetic metabolic diseases.
The committee will consist of nine voting members, including the committee chairperson. Individuals nominated as scientific members must be technically qualified experts in their relevant fields and have experience interpreting complex data.
The committee will 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. Members will be invited to serve for overlapping terms of up to four years.
Subscribe to our e-Newsletters
Stay up to date with the latest news, articles, and events. Plus, get special offers
from American Pharmaceutical Review – all delivered right to your inbox!
Sign up now!