FDA Grants Orphan Drug Status to Papillon Therapeutics' PPL-002 for Danon Disease

Papillon Therapeutics Inc., a clinical-stage biotechnological firm specializing in genetic treatments for inherited diseases, announced that the U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to their experimental therapy, PPL-002, aimed at treating Danon disease.

Carter Cliff, the CEO of Papillon Therapeutics, expressed appreciation for the FDA’s decision, emphasizing the importance of this designation for their pipeline. Cliff highlighted that the therapeutic approach of PPL-002 has potential benefits for treating multiple symptoms and addressing the unmet medical needs of patients suffering from Danon disease and other severe conditions. According to Cliff, this designation confirms the company’s commitment to advancing therapies for rare and debilitating diseases.

The FDA’s Orphan Drug Designation program is designed to support the development of treatments for rare diseases affecting fewer than 200,000 people in the U.S. Benefits for drug developers under this program include tax credits for clinical trial costs and waivers for prescription drug user fees.

PPL-002 is an investigational therapy involving gene-modified CD34+ hematopoietic stem and progenitor cells (HSPCs) for Danon disease treatment. This therapy is intended to produce functional Lamp-2 protein, which is significantly deficient in Danon disease patients. Preclinical studies have shown that PPL-002 can improve disease symptoms in affected tissues. The treatment focuses on addressing multiple organ systems simultaneously, offering the potential to modify and reverse the disease's progression. Research into PPL-002 is partially funded by the California Institute for Regenerative Medicine (CIRM). The principal investigators are Dr. Eric Adler, a clinical professor of medicine and medical director of the heart transplant program, and Dr. Stephanie Cherqui, a professor of pediatrics and director of the Gene Therapy Initiative at the University of California, San Diego.

Danon disease is a rare genetic disorder linked to mutations in the LAMP2 gene, causing the accumulation of autophagic vacuoles in cells, mainly in the heart and skeletal muscles. The disease usually manifests in adolescence or early adulthood, presenting a range of serious symptoms, including severe cardiomyopathy, physical impairment, and retinal abnormalities. The progression of Danon disease is rapid, often leading to life-threatening complications such as heart failure and arrhythmias.

The California Institute for Regenerative Medicine (CIRM) plays a pivotal role in accelerating stem cell treatments for patients with unmet medical needs. CIRM collaborates with academia and industry to fast-track the development of promising stem cell technologies, aiming to bring the future of regenerative medicine closer to reality. The organization is one of the largest in the world dedicated to advancing regenerative medicine.

Papillon Therapeutics Inc. is dedicated to developing a range of multi-systemic genetic treatments targeting the fundamental causes of inherited diseases. Their technology platform ensures durable expression of functional proteins throughout the body. Papillon Therapeutics was founded by leading experts in genetic medicine, including Dr. Stephanie Cherqui. The company is focused on transforming the treatment landscape for genetic disorders through innovative scientific research and development.

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