The FDA is creating a new advisory committee made up of nine voting members who will provide insight into potential treatments for genetic metabolic diseases.
The up-and-coming Genetic Metabolic Diseases Advisory Committee will advise on products used to diagnose, prevent or treat inherited metabolic diseases and will be organized within the FDA’s Division of Rare Diseases and Medical Genetics—a division that was established in 2020.
Metabolic conditions disrupt the chemical process that converts food into energy and removes toxins from the body. There are hundreds of different genetic metabolic diseases, most of which are rare and can be life-limiting. Examples include Fabry Disease, Tay-Sachs Disease, Hunter syndrome and Niemann-Pick, among others.
A raft of biotechs have sprung up with the aim of tackling specific genetic metabolic diseases including the likes of Cyclo Therapeutics, 4D Molecular Therapeutics and UniQure.
The FDA is asking for nominations of qualified individuals to serve on the committee. The agency is seeking 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.