Federal Grant Award Summary

The Food and Drug Administration (FDA) Research program (CFDA 93.103) awarded Icahn School of Medicine at Mount Sinai a $5.17 million Project Grant on September 25, 2025, to conduct a Phase 1/2 clinical trial evaluating oral ciclopirox olamine as a treatment for congenital erythropoietic porphyria (CEP), an ultra-rare autosomal recessive genetic disorder affecting heme biosynthesis. The trial will be led by a team of porphyria experts and will assess the safety, tolerability, and efficacy of ciclopirox olamine—a repurposed oral formulation of an FDA-approved topical fungicide—as a pharmacologic chaperone to stabilize and increase activity of the mutated uroporphyrinogen synthase (UROS) enzyme in CEP patients. This approach targets the underlying disease pathogenesis by reducing toxic porphyrin accumulation, hemolysis, transfusion dependency, splenomegaly, and phototoxic cutaneous damage that characterize the disease. The project extends through August 31, 2029.

Mount Sinai established two clinical trial sites through sub-awards: Stanford University (led by Dr. Hetanshi Naik, with Dr. Robert Desnick as the lead principal investigator) will coordinate enrollment, regulatory approval, and patient management at the Stanford site, while The Cleveland Clinic Foundation (led by Dr. Angelika Erwin) will serve as an additional enrolling site with capacity for approximately two trial participants. Both sub-awardees will conduct activities including institutional review board submissions, database development, patient recruitment and enrollment, safety monitoring, regulatory compliance, data collection, and dissemination through manuscript preparation and scientific presentations.