has granted Orphan Drug Designation to Papillon’s PPL-001, an experimental treatment for Friedreich's ataxia. “We are pleased that the FDA has granted Orphan Drug Designation to our program ...
In 2023, the U.S. Food and Drug Administration approved the first medication to treat people with Friedreich’s ataxia who are age 16 or older. Given that Friedreich’s ataxia is a genetic ...