Approval Pathway UncertaintyFDA recommendation for a prospective, randomized, double-blind sham surgery-controlled trial creates major feasibility challenges that can extend timelines and substantially raise development costs for AMT-130.
Evidence Sufficiency ConcernsRegulatory view that early-stage data compared with external natural history controls are insufficient for approval increases the risk that current datasets will not support a regulatory filing.
Safety And Trial Disruption RiskDose-limiting toxicities, including liver enzyme elevations that prompted dosing pauses, heighten clinical risk and could force protocol modifications that delay progress and increase expenses.