Larimar Therapeutics Announces FDA has Removed Partial Clinical Hold for Nomlabofusp Program in Friedreich's Ataxia

Core Viewpoint - The U.S. FDA has lifted the partial clinical hold on Larimar Therapeutics' nomlabofusp (CTI-1601) clinical program, which is being developed for the treatment of Friedreich's Ataxia (FA) [1][2] Group 1: Clinical Development - Nomlabofusp is a novel protein replacement therapy aimed at delivering frataxin to mitochondria, addressing the root cause of FA [1][4] - The FDA's decision followed a review of data from a completed four-week, placebo-controlled Phase 2 dose exploration study, which included 25 mg and 50 mg cohorts [1][2] - The Phase 2 study showed that nomlabofusp was generally well-tolerated, with a predictable pharmacokinetic profile and dose-dependent increases in frataxin levels in skin and buccal cells [2][3] Group 2: Ongoing Studies - An ongoing open-label extension (OLE) study is evaluating the long-term safety and pharmacokinetics of nomlabofusp, initially using a 25 mg dose [3][7] - The company plans to escalate the dose to 50 mg in the OLE study after further characterization of frataxin pharmacodynamics at the 25 mg dose [2][3] - Interim data from the OLE study is expected in the fourth quarter of 2024, with a Biologics License Application (BLA) submission targeted for the second half of 2025 [7] Group 3: Regulatory Designations - Nomlabofusp has received several designations from the FDA, including Rare Pediatric Disease designation, Fast Track designation, and Orphan Drug designation [4][5] - The European Commission has also granted Orphan Drug Designation, and the European Medicines Agency has awarded a PRIME designation [4]