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BIO Submits Comments on FDA Guidance: Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics

September 26, 2022

On Monday, September 26th, BIO submitted comments in response to the FDA’s draft guidance on clinical pharmacology considerations for development of oligonucleotide therapeutics, which provided recommendations on when certain PKPD assessments may be appropriate and which types of assessments should be considered. In the comments submitted, BIO voiced strong agreement with the FDA’s assessment that this modality is rapidly evolving and has strong potential to treat both rare and common diseases. However, BIO noted that oligonucleotide therapeutics represent a wide range of unique and diverse products with key differences in characteristics such as molecular structure, mechanism of action, and formulation/delivery. Therefore, the inclusion of precise and accurate terminology in the final guidance is paramount. In addition to requesting that FDA reconsider the terminology used, BIO also urged FDA to adjust the scope of the guidance to be more inclusive of this wide range of products. BIO also provided a number of line edits requesting clarification on topics such as determining the potential for QT/QTc prolongation and evaluating oligonucleotides in patients with varying levels of renal/hepatic function.

Download Full Comments Below
BIO Comment Letter - FDA-2022-D-0235
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