New FDA Advisory Committee to Explore Genetic Metabolic Disease Drugs
The FDA has announced a new Genetic Metabolic Diseases Advisory Committee (GeMDAC) to help the agency explore the complex issues related to genetic metabolic disease drug development.
GeMDAC will advise the FDA on the safety and effectiveness of human drug and biological products that are 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, including experts on small population trial designs. Committee members will be invited to serve for overlapping terms of up to four years. In addition to the voting members, the committee may include one nonvoting industry representative.
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.
Nominations or applications to serve on GeMDAC may be submitted by accessing the FDA Advisory Committee Membership Nomination Portal or by mail. Nominations should be submitted no later than Feb. 11, 2024.