Papillon Therapeutics Secures Orphan Drug Designation from FDA for PPL-001 in Friedreich’s Ataxia Treatment

Papillon Therapeutics Inc., a clinical-stage biotechnology company advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited disease, announced that the U.S. Food and Drug Administration (FDA) 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 PPL-001,” said Carter Cliff, chief executive officer of Papillon Therapeutics. “This significant milestone underscores our commitment to developing groundbreaking therapies for patients with rare, debilitating diseases such as Friedreich’s ataxia. We believe this therapy has the potential to treat multiple symptoms and address the unmet treatment needs of patients and their families.”

The FDA’s Orphan Drug Designation program grants orphan status to investigational drugs and biologics intended to treat rare diseases or conditions affecting fewer than 200,000 people in the United States. This designation offers various incentives, including tax credits toward the cost of clinical trials and waivers on prescription drug user fees.