How To Build An Endpoint: Spark Designed Testing Maze To Study Rare Vision Loss
In an interview, Spark President Katherine High explains that multi-luminance mobility test used in clinical development of Spark's gene therapy voretigene sprang from 2011 FDA meeting on need to study functional vision in patients with retinal degenerative diseases. Experience highlights need for new endpoints when targeting previously untreated diseases, she says.
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Established efficacy endpoints may not be suited to assessing gene therapies’ clinically meaningful effects in some rare retinal disorders, US FDA says in draft guidance that gives a shout-out to Spark’s development of a functional vision endpoint for voretigene; guidance also includes recommendations on appropriate control groups and use of sham injections.
Scale-up in particular is a challenge and FDA wants to help lower the barrier, CBER Director Peter Marks tells rare disease conference.
CBER Director Peter Marks says the center implemented a lot in a short amount of time for the RMAT pathway.