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Mpox: Development of Drugs and Biological Products; Guidance for Industry

DraftCenter for Drug Evaluation and Research01/19/2023

Description

FDA is issuing this guidance to support sponsors in their development of drugs1for mpox. This guidance provides nonclinical, virology, and clinical considerations for mpox drug development programs, with a focus on recommendations to support initiation of clinical trials.

Scope & Applicability

Product Classes

3
Preventive vaccines

Not addressed in this guidance

Small Molecule Drug

Category of products requiring specific toxicology and virology data

Biological Product

Regulated under section 351(i) of the PHS Act; Virus, therapeutic serum, toxin, vaccine, or protein applicable to prevention or treatment; Alternative regulation category for products meeting device definition

Stakeholders

1
Sponsor

Entity responsible for submitting applications under section 524B

Regulatory Context

Attributes

1
Substantial Evidence of Effectiveness

The legal standard for proving a drug works; Legal standard required for drug approval

Identified Hazards

Hazards

1
Mitochondrial Toxicity

Risk associated with small molecule inhibitors like nucleoside analogues

Related CFR Sections (3)

  • 21CFR314.600§ 314.600 Scope.

    This subpart applies to certain new drug products that have been studied for their safety and efficacy in ameliorating or preventing serious or life-threatening conditions caused by exposure to lethal or permanently disabling toxic biological, chemical, radiological, or nuclear substances. This subpRead full regulation →

  • 21CFR601.90§ 601.90 Scope.

    This subpart applies to certain biological products that have been studied for their safety and efficacy in ameliorating or preventing serious or life-threatening conditions caused by exposure to lethal or permanently disabling toxic biological, chemical, radiological, or nuclear substances. This suRead full regulation →

  • 21CFR314.126§ 314.126 Adequate and well-controlled studies.

    (a) The purpose of conducting clinical investigations of a drug is to distinguish the effect of a drug from other influences, such as spontaneous change in the course of the disease, placebo effect, or biased observation. The characteristics described in paragraph (b) of this section have been develRead full regulation →

Related Warning Letters (9)

  • Good Laboratory Practice (GLP) for Nonclinical Laboratory Studies

    CCIC Huatongwei International Inspection Co., Ltd.

    2025-08-26
  • Good Laboratory Practice (GLP) for Nonclinical Laboratory Studies

    Jiangsu Kerbio Medical Technology Group Co.

    2025-08-26
  • Investigational Device Exemptions (IDE)/Premarket Approval Application (PMA)

    Mid-Link Testing Company, Ltd

    2024-09-11
  • Investigational Device Exemptions (IDE)/Premarket Approval Application (PMA)

    Sanitation & Environmental Technology Institute dba SDWH

    2024-09-11
  • Good Laboratory Practice (GLP) for Nonclinical Laboratory Studies

    Samm Solutions, Inc., d.b.a. BTS Research

    2023-10-31
  • Bioresearch Monitoring Program

    Valley Biosystems

    2022-08-09
  • Good Laboratory Practice (GLP)

    Toxikon Corporation/Labcorp Bedford LLC

    2022-02-22
  • Good Laboratory Practice (GLP) for Nonclinical Laboratory Studies

    University of Kentucky

    2020-05-05
  • Good Laboratory Practice (GLP) for Nonclinical Laboratory Studies

    Steiner Biotechnology, LLC

    2020-04-07

See Also (8)

Mpox: Development of Drugs and Biological Products; Guidance for Industry | Guideline Explorer | BioRegHub