Final FDA guidance on development of oligonucleotide therapeutics

The US Food and Drug Administration (FDA) has announced the availability of a final guidance for industry titled  Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics, replacing the June 2022 draft guidance on this topic.

Oligonucleotide therapeutics are an emerging therapeutic modality with increasing numbers of drugs in development. Oligonucleotides have a unique ability to modulate gene expression, making them an attractive modality to address a significant proportion of therapeutic targets that are not amenable to traditional small molecules or biologics.

The FDA noted that oligonucleotide therapeutics include a wide variety of synthetically modified RNA or RNA/DNA hybrids that are specifically designed to bind to a target RNA sequence to alter RNA expression and/or downstream protein expression. Many antisense and small interfering RNA (siRNA) oligonucleotide therapeutics have been FDA-approved in recent years.