FDA establishes advisory committee for genetic metabolic disease treatments

2023-12-18

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来源: PMLiVE

The US Food and Drug Administration (FDA) has announced the establishment of a new advisory committee to evaluate potential treatments for genetic metabolic diseases.

The Genetic Metabolic Diseases Advisory Committee will advise the FDA on products used for the diagnosis, prevention and treatment of genetic metabolic diseases under the Division of Rare Diseases and Medical Genetics, established in 2020.

Genetic metabolic diseases are conditions that disrupt an individual’s metabolism, which is the mechanism responsible for transferring food into energy and removing toxins from the body.

Most of these types of diseases are rare, including Hurler syndrome, Gaucher disease and Tay-Sachs disease.

The committee will provide FDA-independent, knowledgeable advice and recommendations on technical, scientific and policy issues in relation to medical products for these diseases.

“Drug development for these conditions has unique and complex challenges, therefore few treatments are available to patients,” explained Janet Maynard, director of the FDA’s Center for Drug Evaluation and Research’s (CDER) Office of Rare Diseases, Paediatrics, Urologic and Reproductive Medicine (ORPURM).

Described as “an essential part of the FDA’s work”, “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,” said Patrizia Cavazzoni, director of the CDER.

With nine voting members, including the committee chairperson, the committee will include experts in multiple areas of metabolic genetics, including carbohydrate disorders, amino acid disorders and organic acidemias.

Furthermore, experts in the management of inborn errors of metabolism, small population trial design, translational science, peadiatrics, epidemiology or statistics and related specialties will also be involved.

All individuals nominated as scientific members of the committee must be technically qualified experts in their relevant fields and have experience interpreting complex data.

In addition to this, the committee will also include a consumer representative and an industry representative, while non-federal members will serve as either special government employees or non-voting representatives, following an invitation to serve for up to four years.

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