Description
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Nonclinical Safety Assessment of Oligonucleotide-Based Therapeutics.” FDA is publishing this draft guidance which, when finalized, will provide recommendations on approaches for the nonclinical safety evaluation of oligonucleotide-based therapeutics (ONTs) to support clinical development and marketing of these products. ONTs present unique challenges and opportunities in the nonclinical evaluation of safety that differ in many regards from those appropriate for small molecule drugs or therapeutic proteins.
Scope & Applicability
Product Classes
4Guidance provides recommendations for the nonclinical safety evaluation of these products.; Nonclinical safety assessment of these products; Nonclinical safety assessment of these therapeutics; Nonclinical Safety Assessment of Oligonucleotide-Based Therapeutics
Type of ONT drug product
Type of ONT drug product
Type of ONT drug product
Stakeholders
1Entity responsible for submitting applications under section 524B
Regulatory Context
Attributes
4Species responsive to the clinical candidate or surrogate
Targeting moieties may confer additional species specificity
Prolonged half-life at the site of action
Estimated by normalizing animal dose to body surface area
Identified Hazards
Hazards
5A safety assessment consideration for ONTs.; Potential to bind to nontargeted RNA or DNA sequences
DNA Reactive Impurities in Pharmaceuticals
Risk associated with intended pharmacology or off-target effects
Section XI of the guidance.
Potential adverse effects resulting from intended PD effect
Related CFR Sections (2)
- 21CFR312.81§ 312.81 Scope.
This section applies to new drug and biological products that are being studied for their safety and effectiveness in treating life-threatening or severely-debilitating diseases.Read full regulation →
- 21CFR600.3§ 600.3 Definitions.
As used in this subchapter:Read full regulation →
See Also (8)
- Severely Debilitating or Life-Threatening Hematologic Disorders: Nonclinical Development of Pharmaceuticals Guidance for Industry (Status: Final)
- Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products: Draft Guidance for Industry (Status: Draft)
- IND Submissions for Individualized Antisense Oligonucleotide Drug Products: Administrative and Procedural Recommendations Guidance for Sponsor-Investigators: Draft Guidance for Sponsor-Investigators (Status: Draft)
- Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Chemistry, Manufacturing, and Controls Recommendations, Guidance for Sponsor-Investigators : Draft Guidance for Industry (Status: Draft)
- IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Clinical Recommendations: Draft Guidance for Industry (Status: Draft)
- Questions and Answers on Biosimilar Development and the BPCI Act Guidance for Industry: Guidance for Industry (Status: Final)
- Principles of Premarket Pathways for Combination Products: Guidance for Industry and FDA Staff (Status: Final)
- Sameness Evaluations in an ANDA — Active Ingredients (Status: Draft)