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 Rare Pediatric Disease designation to Papillon’s PPL-001, an experimental treatment for Friedreich's ataxia. PPL-001 has previously received Orphan Drug designation.
“We are thrilled to receive Rare Pediatric Disease designation for PPL-001 and appreciate the FDA’s ongoing support,” said Carter Cliff, chief executive officer of Papillon Therapeutics. “This designation provides an opportunity to receive a priority review voucher that can be redeemed or monetized to accelerate our pipeline. It marks an important milestone in our commitment to making a meaningful impact in the lives of patients and their families.”
The FDA grants Rare Pediatric Disease designation to therapeutics intended to treat serious or life-threatening rare diseases that affect individuals under the age of 18. Products that receive this designation are eligible to receive a priority review voucher (PRV) upon approval. The PRV can either be redeemed for the sponsor to receive priority review for a product’s marketing application, or can be sold or transferred to another sponsor.
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 the California Institute for Regenerative Medicine (CIRM)
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast-track the development of today’s most promising stem cell technologies. CIRM is one of the world’s largest institutions dedicated to helping people by bringing the future of regenerative medicine closer to reality. For more information, go to www.cirm.ca.gov.
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.
