Case Study: Developing a Complex Dosing Drug Schedule for a DART Pompe Disease Study

Amics Therapeutics, based in Cranbury, NJ (USA), focuses on finding treatments for a DART Pompe disease studyrange of devastating and rare and orphan diseases. Its lead biologics program is ATB200/AT2221, a uniquely engineered Pompe disease enzyme replacement therapy that will be administered in combination with a pharmacological chaperone and tested in multiple human genetic diseases.

A Short-Half Life Investigational Drug Suggests an Every-Other-Day Complex Dosing Regime

The Amicus and Covance dialogue began at the 2017 Society of Toxicology conference and resulted in a large series of DART studies being awarded to Covance at its center for biotech excellence in Greenfield, IN. Early in the engagement, Covance’s most experienced US-based DART team member provided consultation on study design to make it more consistent with FDA guidelines and expectations. Continue reading

5 Things to Know About the Evolving Requirements For SEND

As our industry approaches the one year anniversary of the implementation of SEND (Standard for the Exchange of Nonclinical Data) datasets as required by the FDA for regulatory submissions, attention is shifting to the next set of requirements. From recent notices to upcoming compliance dates, we’ve compiled five key highlights for your information that will also help you proactively prepare for the changes. Continue reading