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-002, an experimental treatment for Danon disease. PPL-002 has previously received Orphan Drug designation.
“We are pleased that the FDA has granted a second Rare Pediatric Disease designation to our pipeline,” said Carter Cliff, chief executive officer of Papillon Therapeutics. “This designation offers an additional opportunity to receive a priority review voucher, which will enable us to accelerate our pipeline and better serve patients and families affected by Danon disease.”
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-002
PPL-002 is an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy for the treatment of Danon disease. This therapeutic is designed to express functional Lamp-2 protein, which is critically deficient in people living with Danon disease. In preclinical studies, PPL-002 has been shown to improve disease phenotype in the affected tissues. PPL-002’s treatment approach involves targeting multiple organ systems simultaneously in patients, offering the potential to modify and reverse disease progression. PPL-002 research is funded in part by the California Institute for Regenerative Medicine (CIRM). The principal investigators are Eric Adler, M.D., clinical professor of medicine and medical director of the heart transplant program, and Stephanie Cherqui, Ph.D., professor of pediatrics and director of the Gene Therapy Initiative, at the University of California San Diego.
About Danon Disease
Danon disease is a rare, X-linked genetic disorder caused by mutations in the LAMP2 gene, leading to the accumulation of autophagic vacuoles in cells, primarily in the heart and skeletal muscles. This condition typically manifests in adolescence or early adulthood. Patients experience a range of life-threatening symptoms, including severe cardiomyopathy, physical impairment and retinal abnormalities. Danon disease progresses rapidly, often resulting in life-threatening complications such as heart failure and arrhythmias.
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.
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