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The Luxturna Model: Spark’s Approach To Retinal Disease Endpoint Embraced In Gene Therapy Guidance

 
• By Sue Sutter

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not be suited to assessing gene therapies’ clinically meaningful effects in some rare retinal disorders, US FDA says in draft guidance that gives a shout-out to Spark’s development of a functional vision endpoint for voretigene; guidance also includes recommendations on appropriate control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.

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The US FDA’s clinical development advice for gene therapies to treat retinal disorders openly embraces the type of novel endpoint development that Spark Therapeutics Inc. pursued with Luxturna, the first gene therapy approved in this space.

The draft guidance includes examples of established efficacy endpoints but also encourages sponsors to develop and propose novel endpoints to measure clinically meaningful effects, as Spark did with its

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