FDA sets up new advisory committee on genetic metabolic diseases

The US Food and Drug Administration (FDA) yesterday announced the establishment of a new Genetic Metabolic Diseases Advisory Committee (GeMDAC) to help the agency explore the complex issues related to genetic metabolic disease drug development.

Genetic metabolic diseases are rare genetic conditions that interfere with a person’s metabolism, the body’s ability to convert food into energy and remove waste and unhealthy substances. There are hundreds of genetic metabolic diseases, and many have few or no treatment options.

The GeMDAC will advise the FDA on the safety and effectiveness of human drug and biological products under investigation or submitted for marketing approval for use in the treatment of genetic metabolic diseases. The committee will consist of nine voting members with technical and scientific expertise from diverse disciplines and backgrounds. Committee members will be invited to serve for overlapping terms of up to four years.