Rare Neurodegenerative Disease Grants Program

Rare Neurodegenerative Disease Grants Program is sponsored by FDA Office of Orphan Products Development (OOPD). This program awards grants and contracts to cover costs of research and development of interventions intended to prevent, diagnose, mitigate, treat or cure ALS and other rare neurodegenerative diseases in adults and children.

FDA Rare Neurodegenerative Disease Grants Program | FDA Skip to in this section menu Medical products for rare diseases and conditions Grant Programs to Support Development of Medical Products for Rare Diseases FDA Rare Neurodegenerative Disease Grants Program Grant Programs to Support Development of Medical Products for Rare Diseases Launched when the Accelerating Access to Critical Therapies for ALS (also referred to as ACT for ALS) law was enacted in December 2021, the Rare Neurodegenerative Disease (RNDD) grants program supports research in amyotrophic lateral sclerosis (ALS) and other rare neurodegenerative diseases in adults and children.

Under this program, FDA awards grants and contracts to public and private entities to cover costs of research and development of medical products intended to prevent, diagnose, mitigate, treat or cure ALS and other rare neurodegenerative diseases in adults and children. This program supports research in ALS as well as other rare neurodegenerative diseases. What are neurodegenerative diseases?

Neurodegenerative diseases occur when nerve cells in the brain or peripheral nervous system lose function over time and ultimately die. In general, they are incurable and debilitating conditions and are progressive. The term “rare disease or condition” is defined in 21 U.S.C.

360ee and generally they are considered by FDA as diseases with a prevalence of fewer than 200,000 people in the United States or in the case of an acute disease (e.g., less than one year duration), an annual incidence of fewer than 200,000 per year.

Some examples of what FDA currently considers rare neurodegenerative diseases include ALS, Cockayne syndrome, ataxia telangiectasia, mucopolysaccharidosis (MPS) type III, familial dysautonomia, and Niemann-Pick Type C.

To learn more about what qualifies for the RNDD grants program, please see: FDA Operational Definition of a Rare Neurodegenerative Disease Grants Awarded and Future Opportunities A list of grants awarded under this program is available at: Rare Neurodegenerative Disease Grants Awarded .

OOPD also administers the Orphan Products Grants Program , which fosters development of new medical products for rare diseases and conditions through clinical trials and natural history studies . Contact the OOPD grants team .