Description
The purpose of this guidance is to describe the nonclinical information that FDA recommends to support an investigational new drug application (IND) for an antisense oligonucleotide being developed to treat a severely debilitating or life-threatening (SDLT) disease caused by a unique genetic variant where only a small number of individuals are prospectively identified (usually one or two). The investigational antisense oligonucleotide should be from a well-characterized chemical class for which there is substantial nonclinical information and clinical experience that is publicly available or to which the sponsor-investigator (hereafter referred to as sponsor) has a right of reference.
Scope & Applicability
Product Classes
2Many antisense oligonucleotide therapeutics have been FDA-approved.
Individualized drug products for severely debilitating or life-threatening diseases; Individualized ASO drug products for SDLT diseases
Stakeholders
2Governs top dose in clinical studies
Individuals who both initiate and conduct an investigation
Regulatory Context
Attributes
1Preferable high dose for clinical dose selection flexibility.
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 →
- 21CFR314.105§ 314.105 Approval of an NDA and an ANDA.
(a) FDA will approve an NDA and send the applicant an approval letter if none of the reasons in § 314.125 for refusing to approve the NDA applies. FDA will issue a tentative approval letter if an NDA otherwise meets the requirements for approval under the Federal Food, Drug, and Cosmetic Act, but caRead full regulation →
See Also (7)
- Safety Reporting Requirements for INDs (Investigational New Drug Applications) and BA/BE (Bioavailability/Bioequivalence) Studies: Guidance for Industry and Investigators (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- Safety Reporting Requirements and Safety Assessment for IND and Bioavailability/Bioequivalence Studies: Draft Guidance for Industry (Status: Draft)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)
- Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment Guidance for Industry (Status: Final)
- Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry: Guidance for Industry (Status: Final)
- Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products: Draft Guidance for Industry (Status: Draft)
- Rare Diseases: Considerations for the Development of Drugs and Biological Products (Status: Final)