FDA Office of Orphan Products Development (OOPD)

OOPD is the part of the US Food and Drug Administration that handles rare disease incentives: orphan drug designation, Humanitarian Use Device designation, rare pediatric disease designation, and several grant programs. It sits in the Office of the Chief Medical Officer within the Office of the Commissioner. For prion disease it matters in two ways. Designation could give a prion therapy tax credits, user fee exemption and possible seven years of exclusivity after approval. And its grants, including the rare neurodegenerative stream, are a possible funding route for natural history and trial work. No source read here shows an FDA orphan designation for any prion disease product, but the official database could not be searched, so this is unconfirmed rather than established.

Key facts

  • OOPD supports development of rare disease treatments by deciding whether products qualify for incentives and by funding research through grants.
  • US orphan designation covers drugs and biologics for diseases affecting fewer than 200,000 people in the US; Humanitarian Use Device designation covers devices for fewer than 8,000 people per year.
  • Legal basis is the Orphan Drug Act (Public Law 97-414) and 21 CFR Part 316; benefits are tax credits for qualified trials, exemption from user fees and potential seven year exclusivity after approval.
  • Designation is separate from approval; designated products get the same scientific review as any other drug, and requests go in through the CDER NextGen portal, by email or by mail.
  • The Clinical Trials Grants Program has operated since 1983 and the Natural History Studies Grants Program began in 2016.
  • OOPD also runs the Rare Neurodegenerative Disease Grants Program, created under the ACT for ALS in 2021, with awards since 2022.
  • As of the FDA funding page dated 2026-04-14, open NOFOs were RFA-FD-25-020 (clinical studies, next receipt dates 2026-10-20 and 2027-10-19), RFA-FD-25-017 (natural history studies, next receipt date 2028-02-08) and RFA-FD-25-001 (rare neurodegenerative trials, only a 2025-10-21 date).
  • Ionis lists ION717 (prion disease) as an investigational antisense drug in a Phase 1/2 study; the company's FDA orphan and rare pediatric designation footnote applies to a different drug, obudanersen for Angelman syndrome. An aggregator likewise records no designations for ION717.
  • A March 2026 trade article described orphan designation as a possible future incentive for ION717 rather than a granted one (search excerpt only). A UCL page on PRN100 as read through search does not mention orphan designation.
  • In the EU, by contrast, the EMA register holds two CJD orphan designations: EU/3/17/1883 (NSC500, 2017, already in the atlas) and EU/3/24/2958 granted 2024-07-25 to RegSmart Life Science AB for a small molecule.
  • The RDEA pilot (novel rare disease endpoints) is a joint CDER and CBER program, announced 2022-10-27, admitting at most three proposals a year through FY2027; its page does not name OOPD and lists no accepted programs.

Timeline

  1. 1983Orphan Drug Act enacted and the Clinical Trials Grants Program begins.
  2. 2016Natural History Studies Grants Program begins.
  3. 2021ACT for ALS creates the rare neurodegenerative grants authority run through OOPD.
  4. 2022Federal Register notice announces the RDEA pilot.
  5. 2024EMA grants a CJD orphan designation (EU/3/24/2958) to RegSmart Life Science AB; no US counterpart found.
  6. 2025Receipt date for NOFOs RFA-FD-25-020 and RFA-FD-25-001.
  7. 2026FDA funding opportunities page current as of this date.
  8. 2026Next receipt date for RFA-FD-25-020.

Last checked 1 November 2026

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