FDA takes new steps aimed at advancing development of individualized medicines to treat genetic diseases

Dec. 16, 2021

The U.S. Food and Drug Administration (FDA) is issuing draft guidance for managing the administration of individualized investigational Antisense Oligonucleotide (ASO) drugs in a clinical trial and conducting clinical assessments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The draft guidance describes important clinical considerations for investigational new drug application (IND) submissions to support initial and continued administration, dosing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 The draft guidance is specifically tailored to the unique circumstances involving relatively few (typically one or two) individuals.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 hopeful these draft guidances, once finalized, will help promising drugs reach patients in a timely manner.

FDA release