Spark's Gene Therapy Luxturna Sails Through US FDA Panel
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.
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The first of what promises to be a wave of potentially curative gene therapies are coming to market. One of the challenges for sponsors will be how to deliver long-term outcomes data demanded by regulators and payors from patients who don’t have any reason to go to the doctor.
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not be suited to assessing gene therapies’ clinically meaningful effects in some rare retinal disorders, US FDA says in draft guidance that gives a shout-out to Spark’s development of a functional vision endpoint for voretigene; guidance also includes recommendations on appropriate control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.
Review and approval of first directly administered gene therapy targeting a disease caused by genetic mutations embodies 'spirit' of agency's forthcoming series of guidance documents on gene therapies, FDA Commissioner Gottlieb says; documents are likely to encourage development of the kind of novel and clinically meaningful endpoint that Spark used in its clinical program.