FDA DRAFT GUIDANCE “IND Submissions for Individualized Antisense Oligonucleotide Drug Products: Administrative and Procedural Recommendations Guidance for Sponsor-Investigators”
This guidance is intended for sponsor-investigators developing individualized investigational antisense oligonucleotide (ASO) drug products for a severely debilitating or life-threatening genetic disease. Most often, individuals with such diseases will not have FDA-approved treatment options and their diseases will be rapidly progressing, resulting in early death and/or devastating irreversible morbidity within a short time frame. In these situations, drug development targeted at a larger number of patients with the ASO is not anticipated because of the specificity of the mechanism of action of the ASO drug product combined with the rarity of the treatment-amenable patient population.
The guidance was developed to advise those developing ASO products on an approach to interacting with, and making regulatory submissions to, the FDA. The guidance addresses the following points:
- The approach to obtaining feedback from the FDA,
- The expectations and process for making regulatory submissions to the FDA,
- Recommendations about the requirement for Institutional Review Board (IRB) review of the protocols within, and
- How to obtain informed consent.