Orphatech Pharmaceuticals

Orphatech is a clinical-stage biopharmaceutical company focused on developing innovative therapies for rare orphan diseases, with a primary emphasis on lysosomal storage disorders. The company's lead program, ORP-101, is a novel substrate reduction therapy (SRT) designed to treat Niemann-Pick disease type C (NPC) by reducing the accumulation of toxic lipids. Orphatech aims to improve upon existing treatments by utilizing a gut-restricted approach to minimize systemic side effects while maintaining efficacy. Based in the United Kingdom, the company leverages expertise in rare disease drug development to address significant unmet medical needs in pediatric and adult populations. The company has received Orphan Drug Designation from both the FDA and EMA for its lead candidate.