Drug DesignationsNomlabofusp previously received Rare Pediatric Disease Designation, Fast Track Designation, and Orphan Drug Designation from the FDA, as well as Orphan Drug Designation by the European Commission and PRIME designation by the EMA.
Financial PositionLRMR ended 2024 with $183.5M in cash, cash equivalents, and marketable securities, which provides runway into 2Q26.
Regulatory ApprovalThe FDA is open to considering skin frataxin levels as a potential novel surrogate endpoint for Accelerated Approval.