Spark Therapeutics faces a grilling as FDA ponders a pioneering OK for gene therapy
With one of the most closely-watched FDA panel reviews of the year coming up for Spark Therapeutics’ $ONCE lead gene therapy, internal reviewers at the FDA have raised some serious questions for the experts to consider.
At the top of the heap of considerations the panel experts will field on Thursday are questions about the durability of this eye therapy, the possibility that patients will need multiple treatments in order to preserve vision gains, the right age to use it and the use of a completely novel endpoint for the primary goal when standard visual acuity achievements fell far short of the goal on statistical significance.
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