Drugs / Vatiquinone
Vatiquinone
also known as
Epi-743 · (r)-alpha-tocotrienol quinone · .alpha.-tocotrienol quinone · PTC-743 · Vatiquinona
Small molecule targets NQO1
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Rejected (CRL) | US (FDA) | — | Friedreich ataxia | 2025-08-19 | “FDA has issued a Complete Response Letter (CRL) related to the New Drug Application (NDA) for vatiquinone” ptcbio.com ↗ |
| Filed | US (FDA) | — | Friedreich ataxia | 2025-05-06 | “Vatiquinone NDA for pediatric and adult patients with Friedreich's ataxia accepted and granted Priority Review by the FDA” ptcbio.com ↗ |
| Filed | US (FDA) | — | Friedreich ataxia | 2024-12-19 | “announced today the submission of the vatiquinone New Drug Application (NDA)” ptcbio.com ↗ |
| Filed | Japan (PMDA) | — | Friedreich ataxia | — | “Japan regulatory submission under review and a decision is expected in Q4 2025” ptcbio.com ↗ “Japan NDA review is ongoing with decision expected in Q4 2025” ptcbio.com ↗ |
Trials 14
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT01719523 | Oct 2012 → Oct 2013 | Tourette syndrome | Yale University | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT01642056 | Sep 2012 → Sep 2019 | mitochondrial disease, myopathy, nervous system disorder | National Human Genome Research Institute (NHGRI) | Completed | No outcome recorded |
| Phase 25 trials | ||||||
| Phase 2 | NCT05485987 | Oct 2022 → Aug 2024 | Friedreich ataxia | PTC Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT02226458 | Oct 2014 → Nov 2015 | autism spectrum disorder | Edison Pharmaceuticals Inc | Withdrawn | No outcome recorded Stop: Enrollment |
| Phase 2 | NCT02104336 | Aug 2014 → Nov 2015 | Pearson syndrome | Edison Pharmaceuticals Inc | Terminated | No outcome recorded |
| Phase 2 | NCT02352896 | Jan 2014 → Oct 2023 | Leigh syndrome | PTC Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT01962363 | Oct 2013 → Jun 2016 | Friedreich ataxia | University of South Florida | Completed | No outcome recorded |
| Phase 2/32 trials · 2 missed | ||||||
| Phase 2/3 | NCT04577352 | Dec 2020 → Apr 2023 | Friedreich ataxia | PTC Therapeutics | Completed | Missed primary |
| Phase 2/3 | NCT04378075 | Sep 2020 → Mar 2023 | Leigh syndrome, MELAS syndrome, epilepsy, mitochondrial DNA depletion syndrome 4a +1 | PTC Therapeutics | Terminated | Missed primary Stop: Business |
| Phase 33 trials · 1 missed | ||||||
| Phase 3 | NCT07681713 | Aug 2026 → Mar 2029 expected | Friedreich ataxia | PTC Therapeutics | Not yet recruiting | No outcome recorded |
| Phase 3 | NCT05515536 | Dec 2022 → Dec 2027 expected | Friedreich ataxia | PTC Therapeutics | Active not recruiting | Missed primary |
| Phase 3 | NCT05218655 | Jun 2022 → Apr 2025 | mitochondrial disease | PTC Therapeutics | Completed | No outcome recorded |
| Phase not stated2 trials | ||||||
| — | NCT01370447 | — | mitochondrial disease | PTC Therapeutics | No longer available | No outcome recorded |
| — | NCT02300753 | — | Leber hereditary optic neuropathy | Edison Pharmaceuticals Inc | No longer available | No outcome recorded |
| Comparator or background therapy7 trials | ||||||
| Phase 2 | NCT02257983 Comparator | Oct 2014 → Nov 2015 | noise induced hearing loss | Edison Pharmaceuticals Inc | Completed | No outcome recorded |
| Phase 2 | NCT01923584 Comparator | Sep 2013 → Dec 2015 | Parkinson disease | University of South Florida | Completed | No outcome recorded |
| Phase 2 | NCT01793090 Comparator | Jan → Jul 2013 | methylmalonic aciduria and homocystinuria type cblC, retinal disorder | Bambino Gesù Hospital and Research Institute | Completed | No outcome recorded |
| Phase 2 | NCT01822249 Comparator | Jan → Dec 2013 | Rett syndrome | Edison Pharmaceuticals Inc | Completed | No outcome recorded |
| Phase 2 | NCT01728064 Comparator | Dec 2012 → Oct 2015 | Friedreich ataxia | PTC Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT01721733 Comparator | Oct 2012 → Feb 2015 | Leigh syndrome | PTC Therapeutics | Completed | No outcome recorded |
| — | NCT07159139 Background | — | mitochondrial disease | Medical University of South Carolina | No longer available | No outcome recorded |
News releases announcing trial results or a regulatory action · 6
| Date | Issuer | Release |
|---|---|---|
| 2025-08-19 | PTC Therapeutics | Regulatory PTC Therapeutics Receives Complete Response Letter for Vatiquinone NDA ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) related to the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich's ataxia. |
| 2025-02-19 | PTC Therapeutics | Regulatory PTC Therapeutics Announces FDA Acceptance and Priority Review for Vatiquinone NDA for the Treatment of Children and Adults with Friedreich's Ataxia ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich's ataxia (FA). |
| 2024-12-19 | PTC Therapeutics | Regulatory PTC Therapeutics Announces Vatiquinone NDA Submission to FDA for the Treatment of Children and Adults Living with Friedreich Ataxia ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the submission of the vatiquinone New Drug Application (NDA) for the treatment of children and adults living with Friedreich ataxia (FA) to the U.S. Food and Drug Administration (FDA). |
| 2024-10-08 | PTC Therapeutics | Results PTC Therapeutics Announces Positive Results from Long-Term Treatment Studies and Updates on Regulatory Progress for Vatiquinone Friedreich Ataxia Program ptcbio.com ↗
The pre-specified endpoint for two different FA long-term extension studies was met, with highly statistically significant evidence of durable treatment benefit on disease progression. |
| 2023-06-29 | PTC Therapeutics | Results PTC Therapeutics Announces Results from MIT-E Clinical Trial of Vatiquinone for the Treatment of Mitochondrial Disease Associated Seizures ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the MIT-E trial of vatiquinone for the treatment of mitochondrial disease associated seizures (MDAS) failed to achieve its primary endpoint of reduction in observable motor seizures. |
| 2023-05-23 | PTC Therapeutics | Results PTC Therapeutics Announces Topline Results from Vatiquinone MOVE-FA Registration-Directed Trial ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today reported topline results from the MOVE-FA trial of vatiquinone in patients with Friedreich ataxia. |
All press releases naming this drug 8 releases
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 22 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Vatiquinone | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05485987 ↗ |
| Known as | (r)-alpha-tocotrienol quinone | ChEMBL registry synonym — accepted as the source's own label CHEMBL1812161 ↗ |
| Known as | .alpha.-tocotrienol quinone | ChEMBL registry synonym — accepted as the source's own label CHEMBL1812161 ↗ |
| Known as | Epi-743 | “Drug: Epi-743” NCT01793090 ↗ |
| Known as | PTC-743 | ChEMBL registry synonym — accepted as the source's own label CHEMBL1812161 ↗ |
| Known as | Vatiquinona | ChEMBL registry synonym — accepted as the source's own label CHEMBL1812161 ↗ |
| Modality | Small molecule | “EPI-743 is an orally absorbed small molecule” NCT01719523 ↗ |
| Route | Oral | “oral solution” NCT01370447 ↗ |
| Target | NQO1 | “targeting an enzyme NADPH quinone oxidoreductase 1 (NQO1).” NCT01719523 ↗ |