FDA Guidance for Sponsor-Investigators: IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Chemistry, Manufacturing, and Controls Recommendations
Title:
FDA Guidance for Sponsor-Investigators: IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Chemistry, Manufacturing, and Controls Recommendations
Origin/Publisher:
FDA
Content:
The purpose of this guidance is to provide recommendations regarding the chemistry, manufacturing, and controls (CMC) information that should be provided in an investigational
new drug application (IND) submitted by a sponsor-investigator (hereafter referred to as sponsor) developing an individualized antisense oligonucleotide (ASO) drug product for a severely debilitating or life-threatening (SDLT) disease caused by a unique genetic variant where only a small number of individuals are prospectively identified (typically one or two)