Spark's Vision Loss Gene Therapy Raises US FDA Questions About Novel Endpoint
Executive Summary
Multi-luminance mobility testing was developed by firm to measure functional vision across a range of lighting levels in patients with RPE65 mutations; advisory committee also will consider minimum patient age and clinical stage of disease appropriate for subretinal injections with voretigene.
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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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Improvements seen in multi-luminance mobility testing in the voretigene Phase III trial were clinically meaningful, panelists said, endorsing the novel endpoint for the vision loss treatment. They also were persuaded by first-hand accounts from patients.