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The FDA has weighed in on Novartis’ pioneering personalized cancer therapy, which looks almost certain to win approval. But the agency is wading into uncharted territory, bringing along a bevy a questions on manufacturing and long-term safety to a hotly anticipated public forum.

In briefing documents released before a Wednesday hearing on Novartis’ treatment, FDA staff made clear that it has little doubt the therapy is effective in treating advanced cases of leukemia in children and young adults. Of greater concern is whether Novartis can reliably produce and deliver the treatment, and whether the children and young adults who receive it stay healthy years after dosing.

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