Drugs / Eladocagene Exuparvovec
Eladocagene Exuparvovec
English name eladocagene exuparvovec
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed aromatic L-amino acid decarboxylase deficiency 2 milestones | |||||
| Filed | US (FDA) | Upstaza | aromatic L-amino acid decarboxylase deficiency | 2024-05-14 | “the FDA has accepted for filing the Biologics License Application (BLA) for Upstaza™” ptcbio.com ↗ |
| Filed | US (FDA) | Upstaza | aromatic L-amino acid decarboxylase deficiency | 2024-03-19 | “today announced the submission of a BLA to the U.S. FDA for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency” ptcbio.com ↗ |
| Clinical hold Parkinson disease 1 milestone | |||||
| Clinical hold | US (FDA) | — | Parkinson disease | 2020-12-22 | “FDA has notified Neurocrine Biosciences (Nasdaq: NBIX) that it has placed a clinical hold on the RESTORE-1 clinical trial of NBIb-1817 (VY-AADC).” voyagertherapeutics.com ↗ “The RESTORE-1 Phase 2 clinical trial for VY-AADC (NBIb-1817) remains on FDA clinical hold.” voyagertherapeutics.com ↗ |
| Approved Indication not stated 1 milestone | |||||
| Approved | EU (EMA) | Upstaza | — | 2022-07-18 | europa.eu ↗ |
Trials 5
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 12 trials · 1 met primary | ||||||
| Phase 1 | NCT07267065 | Feb 2026 → Aug 2028 expected | young-onset Parkinson disease | Krzysztof Bankiewicz | Not yet recruiting | No outcome recorded |
| Phase 1 | NCT02852213 | Jul 2016 → Mar 2029 expected | aromatic L-amino acid decarboxylase deficiency | Krzysztof Bankiewicz | Recruiting | Met primary |
| Phase 1/21 trial · 1 met primary | ||||||
| Phase 1/2 | NCT01395641 | Oct 2014 → Dec 2020 | aromatic L-amino acid decarboxylase deficiency | National Taiwan University Hospital | Completed | Met primary |
| Phase 22 trials | ||||||
| Phase 2 | NCT04903288 | May 2021 → May 2023 overdue | aromatic L-amino acid decarboxylase deficiency | PTC Therapeutics | Active not recruiting | No outcome recorded |
| Phase 2 | NCT02926066 | Nov 2016 → Jan 2022 | aromatic L-amino acid decarboxylase deficiency | National Taiwan University Hospital | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 14
| Date | Issuer | Release |
|---|---|---|
| 2024-11-13 | PTC Therapeutics | Regulatory PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy ptcbio.com ↗ |
| 2024-05-14 | PTC Therapeutics | Regulatory PTC Therapeutics Announces FDA Acceptance and Priority Review of the BLA for Upstaza™ ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the FDA has accepted for filing the Biologics License Application (BLA) for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of AADC deficiency. |
| 2024-03-19 | PTC Therapeutics | Regulatory PTC Therapeutics Provides Key Regulatory Updates ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the submission of a BLA to the U.S. FDA for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. |
| 2022-11-17 | PTC Therapeutics | Regulatory Upstaza™ Gene Therapy Granted Marketing Authorization for AADC deficiency by UK's MHRA ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Upstaza™ (eladocagene exuparvovec) was granted authorization by the Medicines and Healthcare Products Regulatory Agency (MHRA) in Great Britain. |
| 2022-07-20 | PTC Therapeutics | Regulatory Upstaza™ Granted Marketing Authorization by European Commission as First Disease-Modifying Treatment for AADC Deficiency ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Upstaza™ (eladocagene exuparvovec) was granted marketing authorization by the European Commission. |
| 2022-05-20 | PTC Therapeutics | Regulatory PTC Therapeutics Receives Positive CHMP Opinion for Upstaza™ for the Treatment of AADC Deficiency ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Upstaza™ (eladocagene exuparvovec; PTC-AADC) received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). |
| 2021-09-29 | PTC Therapeutics | Results Results Show Long-Lasting and Holistic Improvements in Children with AADC Deficiency Treated with PTC-AADC Gene Therapy ptcbio.com ↗
The new, five-year results show that motor function improvements were sustained, demonstrating that the treatment effect of PTC-AADC is durable, which is highly important for a single administration therapy for a chronic, fatal disease. |
| 2021-08-23 | PTC Therapeutics | Results Targeting the Putamen with Gene Therapy Leads to Sustained Improvements in Motor and Non-Motor Functions in Children with AADC Deficiency ptcbio.com ↗
The data reported in this article show that the surgical approach of delivering our novel PTC-AADC gene therapy directly to the putamen robustly produces dopamine in the brain that results in sustained and substantial functional improvements in children with AADC deficiency. |
| 2020-12-22 | Voyager Therapeutics | Regulatory Voyager Therapeutics Provides Update on NBIb-1817 (VY-AADC) Gene Therapy Program voyagertherapeutics.com ↗
Voyager Therapeutics, Inc. (Nasdaq: VYGR) today announced that the U.S. Food and Drug Administration (FDA) has notified Neurocrine Biosciences (Nasdaq: NBIX) that it has placed a clinical hold on the RESTORE-1 clinical trial of NBIb-1817 (VY-AADC). |
| 2017-09-06 | Voyager Therapeutics | Results Voyager Therapeutics Announces Positive Results from Ongoing Phase 1b Trial of VY-AADC01 for Advanced Parkinson’s Disease voyagertherapeutics.com ↗
The results demonstrated durable, dose-dependent and time-dependent improvements across multiple measures of patients’ motor function after a one-time administration of the gene therapy. |
| 2017-04-25 | Voyager Therapeutics | Results Voyager Therapeutics Announces Additional Results from Phase 1b Trial of VY-AADC01 for Advanced Parkinson’s Disease voyagertherapeutics.com ↗
Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene therapy company developing life-changing treatments for severe diseases of the central nervous system (CNS), today announced dose-dependent improvements in functional and quality of life measures from the Phase 1b trial of VY-AADC01 for advanced Parkinson’s disease presented as part of an emerging (late-breaking) oral presentation at the AAN Annual Meeting. |
| 2017-01-20 | Voyager Therapeutics | Results Voyager Therapeutics Announces Updates from Phase 1b Trial of VY-AADC01 for Advanced Parkinson’s Disease voyagertherapeutics.com ↗
The neurosurgeons are clearly gaining experience administering VY-AADC01 as evident by the increase in surgical coverage of the putamen achieved in Cohort 3 of 42% compared to 34% in Cohort 2 with similar infusion volumes. |
| 2016-12-07 | Voyager Therapeutics | Results Voyager Therapeutics Announces Positive Interim Results from Phase 1b Trial of VY-AADC01 for Advanced Parkinson’s Disease voyagertherapeutics.com ↗ |
| 2016-06-22 | Voyager Therapeutics | Results Voyager Therapeutics Announces Interim Phase 1b Surgical Results for VY-AADC01 in Advanced Parkinson’s Disease voyagertherapeutics.com ↗ |
All press releases naming this drug 14 releases
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 8 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | eladocagene exuparvovec | “Twenty-six patients without head control received bilateral intraputaminal infusions of a recombinant adeno-associated virus type 2 vector containing the human aromatic L-amino...” PMID 34763085 ↗ Nov 2021 |
| Known as | AAV2-hAADC | “We previously reported compassionate use of an adeno-associated virus (AAV) vector containing the human AADC gene (AAV2-hAADC) in four children with AADC deficiency (aged 4-6 years).” PMID 30169182 ↗ Oct 2017 |
| Known as | AGIL-AADC | ChEMBL registry synonym — accepted as the source's own label CHEMBL4298189 ↗ |
| Known as | Eladocagen exuparvovec | ChEMBL registry synonym — accepted as the source's own label CHEMBL4298189 ↗ |
| Known as | Kebilidi | ChEMBL registry synonym — accepted as the source's own label CHEMBL4298189 ↗ |
| Known as | Upstaza | ChEMBL registry synonym — accepted as the source's own label CHEMBL4298189 ↗ |
| Action | Restore | “Demonstration of effective restoration of AADC function” NCT02852213 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “Gene therapy with adeno-associated virus (AAV) serotype 2 (AAV2) driven human AADC (hAADC) has been tested in both animal models and Phase I clinical trials of Parkinson disease.” NCT01395641 ↗ |
| Route | Other | “AAV2-hAADC viral vector will be injected into bilateral putamen by stereotactic surgery.” NCT01395641 ↗ |