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  1. Medical products for rare diseases and conditions

FDA Rare Neurodegenerative Disease Grant Program

FDA launched the FDA Rare Neurodegenerative Disease Grant Program when the Accelerating Access to Critical Therapies for ALS was enacted. The agency 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. 

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.

Future opportunities 

The FDA remains committed to supporting medical product development for all rare neurodegenerative diseases. The agency is continuing to develop additional grant and contract opportunities to meet the intent of Section 5 of the ACT for ALS. See funding opportunities for FDA’s current grant opportunities for rare disease research.

Contact us

  • Questions (including program, scientific/research, and budget): OOPD_RNDDGrants@fda.hhs.gov
  • Technical application submission questions: eRA Service Desk 
  • FDA’s Office of Orphan Products Development main number: 301-796-8660

Additional resources

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