FDA Center for Biologics Evaluation and Research (CBER)

CBER is the unit of the US Food and Drug Administration that regulates biologics for human use: blood and blood components, plasma-derived products, vaccines, allergenics, human tissues, cell therapies and gene therapies. For prion disease it matters in three ways. It wrote the national donor deferral guidance on CJD and variant CJD for blood and plasma, first issued in 1999 and last revised in May 2022. It ran, until 2016, the advisory committee on transmissible spongiform encephalopathies that fed that policy. And its Office of Therapeutic Products (OTP) is the home for gene and cell therapy applications and for its Type C meeting route for new surrogate endpoints, which would apply to a gene-editing or other gene therapy for prion disease. Antisense drugs and antibodies are generally outside this remit (the atlas has not checked which center would take an ASO; FDA sources opened here do not say). Information only, not regulatory or medical advice.

Key facts

  • CBER oversees biologics under the Public Health Service Act and the Federal Food, Drug, and Cosmetic Act; its listed categories are allergenics, blood and plasma-derived products, blood safety devices and tests, gene therapy, human tissues and cellular products, vaccines and xenotransplantation products.
  • It issued the May 2022 CJD and vCJD blood guidance, whose history section dates FDA's first recommendation (a memorandum to blood establishments on deferring recipients of cadaveric pituitary growth hormone) to 1987 and the first CJD and vCJD guidance to 1999.
  • The 2020 revision removed or relaxed several screening recommendations (most European geographic exposure, certain transfusion countries, hGH history, relatives with CJD, bovine insulin) but left UK, France and Ireland deferrals in place until the 2022 revision.
  • FDA's TSE Advisory Committee, set up in 1995, was removed from the list of standing advisory committees by a final rule published on 9 June 2016, because its workload had fallen, it met rarely and the cost was no longer justified.
  • FDA says topics formerly handled by the TSE committee now go to its other advisory committees, naming the Blood Products Advisory Committee as an example; the rule says temporary subject experts can be added.
  • The CBER vaccine page records that in April 2000 CBER asked manufacturers not to use ruminant-derived material from Europe, and that a joint advisory committee on 27 July 2000 judged vCJD risk from vaccines theoretical and remote; it states that no US-licensed vaccine uses bovine material from USDA-listed countries and that no vCJD case has been linked to a vaccine.
  • On plasma-derived products, the US Public Health Service view on the CBER page is that vCJD risk from US-licensed factor VIII is most likely extremely small though not known with certainty, and likely as small or smaller for other plasma derivatives; the page was current as of February 2018.
  • For human cells, tissues and cellular and tissue-based products, federal regulation 21 CFR 1271.75 requires donor screening for human transmissible spongiform encephalopathy including CJD, alongside HIV, hepatitis B and C and syphilis.
  • CBER reorganized its Office of Tissues and Advanced Therapies into the Office of Therapeutic Products, scheduled to start on 26 February 2023, with six offices including one for plasma protein therapeutics manufacturing review.
  • A CBER draft guidance of April 2024 on human and animal derived materials in cell and gene therapy manufacturing lists TSE agents among adventitious agent risks and recommends blood-derived materials come from establishments following the 2022 CJD guidance.
  • CBER published a final guidance on 30 January 2024 on human gene therapy products incorporating genome editing, which finalized a March 2022 draft and is not specific to any disease.
  • OTP offers a Type C meeting on a new surrogate endpoint: a request with preliminary human data, an answer in 21 days, a meeting or written response within 75 days, and no guarantee of acceptance of the surrogate.
  • A search summary describes the Office of Blood Research and Review as the CBER office for transfusion blood components; this was not confirmed on a CBER page.

Timeline

  1. 1987FDA memorandum to blood establishments first recommends deferral of recipients of cadaveric pituitary growth hormone.
  2. 1995TSE Advisory Committee established.
  3. 1999First FDA guidance on CJD and vCJD and blood donation.
  4. 2000Joint FDA advisory committee meeting on 27 July judges vCJD risk from vaccines theoretical and remote.
  5. 2016TSE Advisory Committee terminated, 9 June.
  6. 2020CJD and vCJD guidance revised, relaxing most geographic and several other deferrals.
  7. 2022Current CJD and vCJD blood guidance issued by CBER.
  8. 2023Office of Therapeutic Products scheduled to begin on 26 February.
  9. 2024Final genome editing gene therapy guidance published on 30 January.
  10. 2024Draft guidance on human and animal derived materials for cell and gene therapy.
  11. 2024OTP Type C surrogate endpoint meeting page last updated, 22 August.

Last checked 1 November 2026

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