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FDA Issues Precision Genomic Drug Guidance

DNA analysis graphic

(Gerd Altmann, Pixabay)

4 Jan. 2021. The U.S. Food and Drug Administration issued draft administrative and procedural guidelines for genetic disorders treated with precise gene therapies. The document covers the mechanics for interacting with FDA, particularly for academic scientists who do not normally deal with the agency, when developing individualized biologic products for severely debilitating or life-threatening genetic diseases.

FDA says the document is written for developers of individualized treatments using antisense oligonucleotide therapies. Antisense oligonucleotides are short DNA or RNA sequences for altering RNA, thereby reducing, modifying, or restoring expression of proteins. These treatments are often designed for rare neurological conditions, and in some cases configured for individual patients, often called “N of 1” therapies, to head off severe, rapidly progressing, and life-threatening diseases.

In an editorial appearing on 24 Oct. 2019 in New England Journal of Medicine, Janet Woodcock and Peter Marks, then directors respectively of the Center for Drug Evaluation and Research and Center for Biologics Evaluation and Research at FDA, spell out the scope of the problem. Woodcock and Marks discuss advances in genomics making possible individualized therapies with antisense oligonucleotides, but also highlight practical and ethical concerns for both developers and regulators.

Process leading to IND

FDA’s document aims to provide developers of these precision therapies with a roadmap for presenting their evidence for requesting clearnace to begin human clinical trials. That roadmap recommends making early contact with FDA, particularly when the disease is progressing rapidly, and even highlights best practices for communicating with the agency.

Most of the document outlines the process for an investigational new drug application, or IND, for an individualized antisense oligonucleotide therapy. The process begins with a pre-IND meeting with the agency officials, and materials needed for that meeting. For the application itself, the document highlights the information needed by reviewers, such as evidence from preclinical studies, proposed chemistry and manufacturing of the therapy, and potential safety concerns. In addition, the document calls for addressing ethical questions, such as an institutional review board and gaining informed consent.

FDA acknowledges the guidance document is just a beginning. An agency statement, attributed to acting Center for Drug Evaluation director Patrizia Cavazzoni, says “If we have the scientific ability to develop drug products for these rare diseases, we need to find a way to bring them to patients while ensuring there is the right balance of risk to benefit. This guidance, which provides clarity on the early development and IND submission process, is the FDA’s first step in working with those who are developing these individualized drug products.”

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