RARE Daily

FDA Issues Guidances on N-of-1 ASO Therapies

December 8, 2021

Rare Daily Staff

The U.S. Food and Drug Administration issued two draft guidances for sponsor-investigators developing individualized antisense oligonucleotide (ASO) drug products for a rapidly progressive, severely debilitating, or life-threatening genetic diseases.

The first guidance pertains to the clinical recommendations and covers ethical and human subject protection, diagnostic, and genetic, dosing, administration, safety, and assessment of clinical response to treatment. It also addresses important information to include in IND submissions for ASO drug products for a small number of participants.

The second guidance covers chemistry, manufacturing, and controls (CMC) recommendations information needed to support an investigational new drug application submitted by a sponsor-investigator developing an individualized ASO drug product for a severely debilitating or life-threatening disease caused by a unique genetic variant affecting a small number of individuals (typically one or two).

“Progress in individualized medicines provides hope to patients with severely debilitating or life-threatening genetic diseases,” said acting FDA Commissioner Janet Woodcock. “Advances in technology enable targeting a drug to an individual patient’s genes. Single-subject clinical trials—also called ‘N of 1’ trials—focus on evaluating investigational drug products developed for an individual patient.”

Woodcocks said the field of N of 1 trials is evolving rapidly, but that many of these studies are carried out by academic investigators who many not have much experience interacting with the agency. The FDA took initial steps to provide draft guidance to investigators carrying out such studies earlier this year.

The agency is accepting comments on the draft guidances until February 7, 2022.

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