Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Odiparcil · 1 trial · 1 indication
Changes in physical examination and vital signs
Change from baseline in laboratory safety tests (coagulation, liver enzymes and crystalluria) 12-lead-ECG and bone biomarkers.
Incidence of AEs/SAEs, patient withdrawals from study due to AEs/SAEs,
Change from Baseline in ECG
| Arm | Type | Description |
|---|---|---|
| Double-blind - odiparcil 1000 mg per day | EXPERIMENTAL | 2 tablets of odiparcil 250 mg per os, twice daily (BID) |
| Double-blind - odiparcil 500 mg per day | EXPERIMENTAL | 1 tablet of placebo and 1 tablet of odiparcil 250 mg per os, twice daily (BID) |
| Double-blind - placebo | PLACEBO_COMPARATOR | 2 tablets of placebo per os, twice daily (BID) |
| Open Label - odiparcil 1000 mg per day | EXPERIMENTAL | 2 tablets of odiparcil 250 mg per os, twice daily (BID) |
| Name | Type | Description |
|---|---|---|
| Odiparcil | DRUG | Investigational product: odiparcil 250 mg tablets |
| Placebo | OTHER | Comparator: placebo tablets similar to odiparcil 250 mg tablets |
1. Male or female gender. 2. Age ≥16 years. 3. Diagnosis of MPS VI, demonstrated by a reduced Arylsulfatase B (ARSB) activity relative to the normal range of the laboratory performing the assay in either white blood cells or fibroblast culture or confirmation of two known disease causing mutations i...
Odiparcil is an investigational small molecule being developed for the treatment of mucopolysaccharidosis VI (MPS VI), a rare genetic disorder. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.
Odiparcil is a small molecule designed to address the underlying enzyme deficiency in mucopolysaccharidosis VI. Its specific molecular target is not disclosed in the available information, but it is being studied for its ability to reduce the accumulation of glycosaminoglycans in patients with MPS VI.
Odiparcil is being developed by Inventiva S.A., a biopharmaceutical company. The company's American Depository Shares are traded under the ticker symbol IVA on the NASDAQ.
Odiparcil is in Phase 2 clinical development. It has completed one Phase 2 trial and is not yet approved by the FDA or any other regulatory agency. The drug remains investigational and is being evaluated for safety and efficacy in patients with mucopolysaccharidosis VI.
Odiparcil has been studied in one completed Phase 2 clinical trial, identified as NCT03370653. This randomized, double-blind, placebo-controlled study enrolled 20 participants aged 16 years and older with mucopolysaccharidosis VI across France, Germany, Portugal, and the United Kingdom.
Odiparcil is not known by any alternative names in the available information. It is a distinct investigational compound being developed specifically for mucopolysaccharidosis VI and is not the same as any other marketed or approved drug.