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odiparcil
- Development stage
- Phase 2
- Lead developer
- Inventiva
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Odiparcil is an orally available small molecule drug belonging to the class of coumarins and derivatives, specifically a beta-d-thioxyloside analog. It was developed as a substrate reduction therapy for mucopolysaccharidosis (MPS) disorders, particularly MPS VI (Maroteaux-Lamy syndrome), but also investigated for MPS I and II. Odiparcil acts by modulating glycosaminoglycan (GAG) synthesis, diverting cellular GAGs into soluble forms that can be excreted in urine, thereby reducing pathological accumulation in tissues. This mechanism offers potential benefits over enzyme replacement therapy by improving tissue penetration and addressing unmet needs in lysosomal storage diseases. The drug has demonstrated antithrombotic activity with reduced bleeding risk and has reached Phase II clinical trials across several indications[1][4][5][6][7][8].
- Other names
- odiparcilo
02
Targets
B4GALT1 (Beta-1,4-galactosyltransferase 1)
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