ORL-1B

ORL-1B is a small molecule drug developed by Orpha Labs AG, primarily investigated for the treatment of biotinidase deficiency, a rare congenital metabolic disorder. The drug has reached Phase 2 clinical development status but does not appear to have received regulatory approval as of March 2025. Its mechanism of action and molecular target are not explicitly detailed in public sources; however, it is being studied within the therapeutic areas of congenital disorders and endocrinology/metabolic diseases[1][2]. The only known clinical trial was an open-label, single-arm Phase 2 study in patients with biotinidase deficiency sponsored by Orpha Labs AG[1].