Description
The purpose of this guidance is to assist sponsors in the clinical development of drugs and biological products for the treatment of acute myeloid leukemia (AML). Specifically, this guidance addresses FDA’s current thinking regarding the overall development program and clinical trial designs for the development of drugs to support an indication of treatment of AML, including indications limited to an individual phase of treatment (e.g., maintenance, transplantation preparative regimen, etc.).
Scope & Applicability
Product Classes
4Requires analytical comparability per ICH Q5E
DLT criteria for chimeric antigen receptor T cells
Post-study salvage treatment
A type of ATMP involving recombinant nucleic acids or viral vectors.
Stakeholders
2Entity responsible for submitting applications under section 524B
Possible to conduct the FIH trial in healthy volunteers
Regulatory Context
Attributes
10Criterion for documenting Complete Remission
Final status including alive and TI, alive and transfusion-dependent, dead, or lost
Duration of platelet transfusion independence post baseline
Duration of Red Blood Cell transfusion independence post baseline
Baseline status for transfusion independence assessment.
Baseline status for transfusion independence assessment.
Variable that assists the transfusion independence assessment
Marrow blasts percentage used for CR response
Abbreviation for Transfusion Independence
Clinical benefit resulting from relief of insufficient hematopoiesis
Identified Hazards
Hazards
2Expected toxicity resulting in complications from cytopenias
Toxic event used as a stopping rule in active AML patients.
Related CFR Sections (2)
- 21CFR312.32§ 312.32 IND safety reporting.
(a) Definitions. The following definitions of terms apply to this section:Read full regulation →
- 21CFR312.64§ 312.64 Investigator reports.
(a) Progress reports. The investigator shall furnish all reports to the sponsor of the drug who is responsible for collecting and evaluating the results obtained. The sponsor is required under § 312.33 to submit annual reports to FDA on the progress of the clinical investigations.Read full regulation →
See Also (8)
- Safety Reporting Requirements for INDs and BA/BE Studies: Guidance for Industry and Investigators (Status: Final)
- Specifications for Preparing and Submitting Electronic ICSRs and ICSR Attachments (Status: Final)
- Sponsor Responsibilities - Safety Reporting Requirements and Safety Assessment for IND and Bioavailability/Bioequivalence Studies: Draft Guidance for Industry (Status: Draft)
- Electronic Submission of IND Safety Reports Technical Conformance Guide : Guidance for Industry (Status: Final)
- Acute Myeloid Leukemia: Developing Drugs and Biological Products for Treatment: Guidance for Industry; Availability (Status: Final)
- FDA Regional Implementation Guide for E2B(R3) Electronic Transmission of Individual Case Safety Reports for Drug and Biological Products (Status: Final)
- Safety Reporting Requirements for INDs (Investigational New Drug Applications) and BA/BE (Bioavailability/Bioequivalence) Studies: Guidance for Industry and Investigators (Status: Final)
- Assessment of Abuse Potential of Drugs (Status: Final)