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On Monday, September 26th, BIO submitted comments in response to the FDA’s draft guidance on clinical pharmacology considerations for development of oligonucleotide therapeutics, which provided recommendations on when certain PKPD assessments may be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.
On Tuesday, January 3rd, BIO submitted comments in response to the Food and Drug Administration’s (FDA) recent draft guidance containing recommendations for sponsors on monitoring growth and evaluating pubertal development in clinical trials which…
BIO and its members appreciate the opportunity to work with FDA to develop and align on approaches that are robust, practical, and expedite patient focused drug development. To further enhance the Guidance, we believe that a few areas would benefit…
On Wednesday, June 22nd, BIO submitted comments on the FDA’s Quality Metrics (QM) Reporting Program. In the comments submitted, BIO stated appreciation for FDA’s intent to support a general shift toward a risk-based approach to regulation by…
On Monday, September 26th, BIO submitted comments in response to the FDA’s draft guidance on clinical pharmacology considerations for development of oligonucleotide therapeutics, which provided recommendations on when certain PKPD assessments may be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.