Comparator And Approval UncertaintyReliance on external natural history data as a comparator requires further regulatory alignment and could complicate approval if regulators view the comparison as insufficient.
Financing RiskLimited cash runway relative to ongoing cash burn creates the need for additional financing, which could dilute investors or disrupt development timelines if not secured.
Safety RiskAnaphylactic reactions observed with nomlabofusp, particularly after treatment interruptions, constitute a serious safety and regulatory concern that could restrict clinical use and affect labeling.