Papillon Therapeutics Receives Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for PPL-001 for the Treatment of Friedreich's Ataxia

October 15, 2024. SAN DIEGO--Papillon Therapeutics Inc., today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to Papillon’s PPL-001, an experimental treatment for Friedreich's ataxia. 

PPL-001 has previously received Orphan Drug designation. PPL-001 is an experimental gene-corrected CD34+ hematopoietic stem and progenitor cell (HSPC) therapy. This therapeutic’s novel approach utilizes targeted excision to correct the GAA repeat expansion in Intron 1 of the FXN gene.