FDA Grants Rare Pediatric Disease Designation to Papillon Therapeutics' PPL-001 for Friedreich's
Papillon Therapeutics Inc., a clinical-stage biotechnology company focused on developing multi-systemic genetic medicines to address the root causes of inherited diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to their experimental treatment, PPL-001, for Friedreich’s ataxia. PPL-001 has previously received Orphan Drug designation, underscoring its potential as a significant therapeutic advancement for this rare disease. Publish Date: 15-10-2024 Source: Papillon Therapeutics Inc. Friedreich ataxia, a rare hereditary condition, initiates its course during childhood and results in a gradual nervous system deterioration, along with motor function impairments. Approximately 25% of individuals affected by Friedreich ataxia experience an unconventional variant where symptoms manifest after age 25. This disorder predominantly affects European, Middle Eastern, or North African individuals and is infrequ...