2 Antisense Oligonucleotide Drug Guidances

FDA has issued two draft guidances for individualized antisense oligonucleotide drug products for severely debilitating or life-threatening diseases. The first draft contains clinical recommendations for sponsors developing such drug products. Its focus is to describe important clinical considerations for IND submissions to support initial and continued administration, dosing, and clinical monitoring of an individual with a severely debilitating or life-threatening disease attributable to a unique genetic variant that may be amenable to an antisense oligonucleotide drug product, FDA says.

The second draft contains chemistry, manufacturing, and controls recommendations including nomenclature, structure, and general antisense oligonucleotide drug substance properties; manufacture; characterization; control of excipients; control of drug substance and drug product; reference standards or materials; container closure systems; and stability.