The FDA is issuing a draft guidance to provide recommendations for managing the administration of individualized investigational Antisense Oligonucleotide (ASO) drugs in a clinical trial and conducting clinical assessments of the safety and response during administration of the investigational ASO drug.
The draft guidance describes important clinical considerations for investigational new drug application (IND) submissions to support initial and continued administration, dosing and clinical monitoring of an individual who is eligible to receive an individualized investigational ASO drug. The draft guidance is specifically tailored to the unique circumstances involving relatively few (typically one or two) individuals.
The FDA is also making available an additional draft guidance that provides recommendations regarding the chemistry, manufacturing and controls information that should be provided in an IND application for certain types of ASO drugs. This includes information regarding drug quality (e.g., chemical structure, manufacturing process and critical quality attributes) and manufacturing guidelines.