FDA Type C Surrogate Endpoint meeting

The Type C surrogate endpoint meeting is an FDA formal meeting that a drug sponsor can request to discuss a biomarker it wants to use as a surrogate endpoint that has not before been the primary basis for approval in that context of use, for accelerated or traditional approval. FDA says the meeting looks at feasibility and gaps in knowledge, not at granting acceptance. Separately, FDA publishes a Table of Surrogate Endpoints required by the 21st Century Cures Act, updated every six months, and a draft 2024 guidance on accelerated approval tells sponsors of novel surrogates to consult FDA early. For prion disease, CSF PrP lowering is the candidate surrogate for PrP-lowering drugs; the atlas records a 2019 Type C meeting where FDA did not accept it as a surrogate, and the FDA table page read shows no prion disease entry. No source opened says FDA has accepted CSF PrP. Information only, not regulatory advice.

Key facts

  • The meeting is meant for programs planning to use a biomarker as a surrogate endpoint not previously the primary basis for approval in the proposed context of use, and it applies to both accelerated and traditional approval.
  • Its stated aim is to assess whether the surrogate is feasible as a primary endpoint and to identify knowledge gaps and ways to close them; further sponsor work and agreement with the review office may still be needed afterwards.
  • FDA expects the request once preliminary clinical data show the biomarker responds to the candidate product at doses that appear generally tolerable.
  • At the CBER Office of Therapeutic Products, the request is an IND amendment (or a pre-submission without an IND), carries a list of objectives and questions, and, unlike other Type C requests, includes the full meeting package with preliminary human data.
  • OTP limits a 60 minute meeting to 10 questions counting sub-questions, describes typical packages as 50 to 100 pages, and will not commit to reviewing more than 250 to 300 pages.
  • OTP timelines run from receipt: a grant or denial within 21 days, the meeting or a written response within 75 days, preliminary responses at least 5 days before a meeting, and a summary within 30 days after it.
  • The OTP page states the meeting does not yield a final decision or guarantee that the surrogate will be accepted, and that the sponsor's own minutes are not reviewed, so silence is not agreement.
  • FDA's Table of Surrogate Endpoints implements section 3011 of the 21st Century Cures Act (FD&C Act section 507), has four tables (adult and pediatric, cancer and non-cancer), is updated every six months, and states that acceptability is case by case.
  • The table page, current as of 29 October 2026, lists Alzheimer disease amyloid beta plaque reduction, Duchenne dystrophin and CSF heparan sulfate in MPS II neurological disease among neurological entries, and no prion disease entry was reported.
  • FDA's surrogate resources hub says surrogates reasonably likely to predict clinical benefit can support accelerated approval, with post-marketing confirmation, and names the Formal Meetings guidance and an FDA paper on new surrogate Type C requests as the key documents.
  • A law firm summary of the December 2024 draft accelerated approval guidance says sponsors using a novel surrogate should consult FDA early, and that confirmatory trials should generally be underway before accelerated approval; the guidance implements FDORA.
  • A trade report says the final Formal Meetings guidance incorporates PDUFA VII agreements and replaces a 22 September 2023 draft, and keeps early surrogate consultation inside Type C; its finalisation date is inconsistent on the page.
  • FDA held a public virtual workshop on novel surrogate endpoints for rare disease on 18 May 2026, tied to a PDUFA VII goal and the RDEA pilot.
  • The atlas records that the 2019 Type C meeting (PIND 141250, 31 October 2019) did not accept CSF PrP lowering as a surrogate and gave advice on assay rigour; the minutes could not be read here, so this rests on existing source R24.

Timeline

  1. 2018FDA surrogate endpoint resources hub page last updated, describing the Type C surrogate meeting.
  2. 2019Type C meeting minutes on CSF PrP as a surrogate (PIND 141250), per the atlas record.
  3. 2023Draft Formal Meetings guidance issued, later replaced by the final version.
  4. 2024OTP page on Type C meetings for new surrogate endpoints last updated.
  5. 2024FDA draft guidance on accelerated approval released (one other firm gives 5 December).
  6. 2026FDA public workshop on novel surrogate endpoints in rare disease.
  7. 2026Surrogate endpoint table page current as of this date.

Last checked 1 November 2026

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