Papillon Therapeutics announced today that the FDA granted Orphan Drug Designation to PPL-001, an experitmental genetic therapy. 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.
PPL-001 uses a gene-correction approach to target the genetic mutation causing FA and is currently in the IND enabling studies stage of development. “We believe this therapy has the potential to treat multiple symptoms and address the unmet treatment needs of patients and their families,” said Carter Cliff, chief executive officer of Papillon Therapeutics.
Research for this approach was funded in part by grants from the California Institute for Regenerative Medicine (CIRM), Friedreich’s Ataxia Research Alliance (FARA), and National Institutes of Health (NIH).