The following quote is attributed to Acting FDA Commissioner Janet Woodcock, M.D.
“Progress in individualized medicines provides hope to patients with severely debilitating or life-threatening genetic diseases. 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.
This field is rapidly evolving, and antisense oligonucleotide drugs are the most advanced in this space. However, many N of 1 trials are carried out by academic investigators who may not have much experience interacting with the FDA. Earlier this year, the FDA took initial steps to provide draft guidance to investigators carrying out this critical work. Today, we are issuing additional draft guidance in this area. Once finalized, this guidance will detail important clinical and production considerations to support applications for these types of clinical trials and drug development programs.
The FDA is hopeful these draft guidances, once finalized, will help promising drugs reach patients in a timely manner. We are optimistic that the development of these individualized drug products may continue to change the landscape for treating rare diseases, and the FDA is committed to providing resources and guidance to those advancing these technologies to treat patients in need.”
- Today, the FDA is issuing a draft guidance to provide recommendations for managing the administration of individualized investigational Antisense Oligonucleotide (ASO) drugs in a clinical trial and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 The draft guidance describes important clinical considerations for investigational new drug application (IND) submissions to support initial and continued administration, dosing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 The draft guidance is specifically tailored to the unique circumstances involving relatively few (typically one or two) individuals.
- The FDA is also making available an additional draft guidance that provides recommendations regarding the chemistry, manufacturing and controls information that should be provided in an IND application for certain types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.
Draft Guidance: IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Clinical Recommendations (https://www.fda.gov/regulatory-information/search-fda-guidance-documents/investigational-new-drug-application-submissions-individualized-antisense-oligonucleotide-drug-0)
Draft Guidance: IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Chemistry, Manufacturing, and Controls Recommendations (https://www.fda.gov/regulatory-information/search-fda-guidance-documents/investigational-new-drug-application-submissions-individualized-antisense-oligonucleotide-drug)
Draft Guidance: IND Submissions for Individualized Antisense Oligonucleotide Drug Products: Administrative and Procedural Recommendations Guidance for Sponsor-Investigators (https://www.fda.gov/regulatory-information/search-fda-guidance-documents/ind-submissions-individualized-antisense-oligonucleotide-drug-products-administrative-and-procedural)
Draft Guidance: Nonclinical Testing of Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases Guidance for Sponsor-Investigators (https://www.fda.gov/regulatory-information/search-fda-guidance-documents/nonclinical-testing-individualized-antisense-oligonucleotide-drug-products-severely-debilitating-or)
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.