SAN DIEGO--(BUSINESS WIRE)--Papillon Therapeutics Inc., a clinical-stage biotechnology company advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited disease, today 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.
About PPL-001
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. More than 95% of individuals with Friedreich’s ataxia have a GAA repeat expansion as the disease-causing mutation, leading to systemic loss of frataxin protein. In preclinical studies, PPL-001 has been shown to improve disease phenotype in the affected tissues. This unique multi-systemic approach to treat patients with Friedreich’s ataxia, in which multiple organ systems are targeted simultaneously, offers the potential to modify and reverse disease progression. PPL-001 research is 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). The principal investigator is Stephanie Cherqui, Ph.D., professor of pediatrics and director of the Gene Therapy Initiative at the University of California, San Diego.
About Friedreich’s Ataxia
Friedreich's ataxia is a rare, inherited neurodegenerative disorder that affects multiple tissues throughout the body including the central nervous system, heart, eyes, skeletal muscle, pancreas and more. Characterized by progressive loss of coordination and muscle strength, Friedreich’s ataxia often leads to difficulties with walking, speech and other motor functions. This condition is caused by mutations in the FXN gene, resulting in reduced production of a vital protein called frataxin. Patients typically begin experiencing symptoms in childhood or adolescence and experience significant disability over time.
About Papillon Therapeutics Inc.
Papillon Therapeutics Inc. is a clinical-stage biotechnology company advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited disease. The company’s technology platform enables durable expression of functional protein throughout the body. Papillon was established by leading experts in genetic medicine, including Stephanie Cherqui, Ph.D. For more information, please visit papillon-tx.com.
