At Stannsoporfin Advisory Committee, Talk Of Additional Studies Stirs Feasibility Doubts And High Emotions
InfaCare’s jaundice drug needs more neurodevelopmental outcome studies, US FDA committee says, but one panelist doubts feasibility of long-term follow-up; talk of additional studies leads to an unusual tongue-lashing from one of InfaCare's experts, who says he was 'stunned' by the committee’s conclusion that efficacy had not been shown.
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Uncertainties about clinical benefit and neurodevelopmental safety drive lopsided recommendation against approval; some advisory committee members say that even FDA’s REMS proposal wouldn't be enough to allow stannsoporfin to come to market without further studies.
Whether the single pivotal trial provides ‘substantial and highly persuasive evidence of effectiveness’ is a key question for advisory committee, although FDA also seeks advice on adequacy of the data to characterize adverse neurodevelopment outcomes; if approved, the heme oxygenase inhibitor could face a restrictive Risk Evaluation and Mitigation Strategy.
With inspectors no longer out in the field, they have more time to wrap up work on previously completed facility evaluations, attorney Howard Sklamberg says; inspections freeze could delay manufacturers’ abilities to bring facilities into compliance following adverse inspection findings, as well as agency reclassifications of facility status.