Gene Therapy Guidances Will Focus On Specific Diseases, Gottlieb Says
US FDA's first such guidance will focus on hemophilia, commissioner tells Congress.
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Although a sponsor may conclude that a long-term follow-up protocol is not necessary for a gene therapy, FDA may have a different interpretation, and the agency won't let studies start if that's the case, draft gudiance states.
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not be suited to assessing gene therapies’ clinically meaningful effects in some rare retinal disorders, US FDA says in draft guidance that gives a shout-out to Spark’s development of a functional vision endpoint for voretigene; guidance also includes recommendations on appropriate control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.
Accelerated approval would help agency monitor gene therapies for durability and off-target effects, US FDA commissioner says.