Product Description
PTC Therapeutics is developing Vatiquinone as a treatment for Friedreich Ataxia. (Sourced from: https://clinicaltrials.gov/study/NCT04577352)
Mechanisms of Action: Glutathione Synthesis Modulator
Novel Mechanism: Yes
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: None *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: PTC Therapeutics
Company Location: SOUTH PLAINFIELD NJ 07080-2449
Company CEO: Stuart W. Peltz
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Australia, Brazil, Canada, France, Germany, Italy, Japan, New Zealand, Poland, Spain, Sweden, United Kingdom, United States
Active Clinical Trial Count: 11
Highest Development Phases
Phase 3: Cerebellar Ataxia|Epilepsy|Friedreich Ataxia|Mitochondrial Diseases
Phase 2: Leigh Disease
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| PTC743-NEU-003e-FA | P3 |
Unknown Status |
Friedreich Ataxia |
2027-12-31 |
|
| PTC743-NEU-003e-FA | P3 |
Enrolling by invitation |
Friedreich Ataxia|Cerebellar Ataxia |
2027-12-31 |
|
| 2022-000375-39 | P3 |
Active, not recruiting |
Unknown |
2025-06-13 |
|
| jRCT2011230045 | P3 |
Not yet recruiting |
Mitochondrial Diseases |
2025-03-31 |