Published on 8/13/2019 1:04:00 PM
FDA Announces Availability of a Public Docket, "Evaluating the Clinical Pharmacology of Oligonucleotide Therapeutics"
On August 7, 2019, the US Food and Drug Administration (FDA) announced the availability of a public docket entitled "Evaluating the Clinical Pharmacology of Oligonucleotide Therapeutics." This docket was established to collect comments to aid in developing recommendations for the design and conduct of studies important to the safe and effective use of oligonucleotide therapeutics and facilitate the regulatory assessment of such studies.
Oligonucleotide therapeutics typically are synthetically modified single- or double-stranded ribonucleic acid (RNA) or deoxyribonucleic acid (DNA) that exert pharmacologic effects through a variety of mechanisms (e.g., altered splicing, RNA interference). Compared to small molecule or biological products, oligonucleotide therapeutics have unique characteristics regarding their chemistry, pharmacology, sites of action, pharmacokinetic disposition, and pharmacodynamics. As a result, there may be special considerations for the design and conduct of clinical pharmacology studies to assess oligonucleotide therapeutics, such as those designed to evaluate the effects of organ impairment or drug interactions. None of the currently published FDA guidance documents on clinical pharmacology assessments contain specific recommendations for oligonucleotide therapeutics.
Interested persons are invited to provide detailed information and comments on certain aspects of the evaluation of the clinical pharmacology of oligonucleotide therapeutics. This request focuses on oligonucleotide therapeutics designed to hybridize to a cognate RNA to elicit a pharmacologic effect. FDA is particularly interested in responses to overarching questions regarding the characterization of the effects of hepatic and renal impairment, drug-drug interactions, and immunogenicity on the pharmacokinetics of oligonucleotide therapeutics, as well as the effects of oligonucleotide therapeutics on cardiac electrophysiology. With regard to these four areas, FDA is also requesting comments on what scientific findings may be applied across the sponsor's oligonucleotide therapeutics platform with shared characteristics (e.g., similar backbone modifications) in situations when a sponsor seeks to rely on previously generated data and information that it owns or to which it has a right of reference.
The "Evaluating the Clinical Pharmacology of Oligonucleotide Therapeutics" public docket is available at https://go.usa.gov/xyJ6H. Please refer to the public docket for more details.
FDA established this public docket to collect public comments. FDA will consider all information and comments submitted. You may submit your comments to this public docket by October 7, 2019 to the Docket No. FDA-2019-N-3369 available at https://www.regulations.gov. Your comments do make a difference and can impact the outcomes of FDA regulatory policy. Share your knowledge and experience, and make your voice count.
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This communication was prepared by Office of Clinical Pharmacology, Office of Translational Sciences, CDER, FDA.