Drugs / DUVALGAGENE OTIPARVOVEC
DUVALGAGENE OTIPARVOVEC
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Clinical hold | US (FDA) | — | Fabry disease cardiomyopathy | 2024-08-08 | “U.S. Food and Drug Administration (FDA) removed clinical hold on the Phase 1/2 INGLAXA study for 4D-310 in Fabry disease cardiomyopathy” 4dmoleculartherapeutics.com ↗ “FDA subsequently put the program on clinical hold” 4dmoleculartherapeutics.com ↗ |
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT05629559 | Oct 2022 → Jun 2026 overdue | Fabry disease | 4D Molecular Therapeutics | Recruiting | No outcome recorded |
| Phase 1/2 | NCT04519749 | Sep 2020 → Jan 2026 overdue | Fabry disease | 4D Molecular Therapeutics | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 10
| Date | Issuer | Release |
|---|---|---|
| 2024-08-08 | 4D Molecular Therapeutics | Regulatory 4DMT Reports Second Quarter 2024 Financial Results and Operational Highlights 4dmoleculartherapeutics.com ↗
U.S. Food and Drug Administration (FDA) removed clinical hold on the Phase 1/2 INGLAXA study for 4D-310 in Fabry disease cardiomyopathy |
| 2024-05-09 | 4D Molecular Therapeutics | Results 4DMT Reports First Quarter 2024 Financial Results and Operational Highlights 4dmoleculartherapeutics.com ↗
Announced positive interim data for 4D-310 in Fabry disease cardiomyopathy, demonstrating durable responses on cardiac endpoints and promising cardiac biopsy results |
| 2024-02-12 | 4D Molecular Therapeutics | Results 4DMT Presents Interim Data from 4D-310 INGLAXA Phase 1/2 Clinical Trials for Fabry Disease Cardiomyopathy at WORLDSymposium™ 2024 4dmoleculartherapeutics.com ↗
4D-310 demonstrated clinically meaningful cardiac endpoint improvements through 12-24 months in contractility (echocardiography), peak VO 2 (cardiopulmonary exercise testing) and/or cardiac quality of life (KCCQ) in all five evaluable patients |
| 2023-10-30 | 4D Molecular Therapeutics | Regulatory 4DMT Gains Alignment with FDA on Plan to Lift Clinical Hold on Phase 1/2 INGLAXA Clinical Trial for 4D-310 for Fabry Disease Cardiomyopathy 4dmoleculartherapeutics.com ↗
4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company), a clinical-stage genetic medicines company with three novel, highly targeted next generation AAV vectors currently in the clinic, today announced alignment with the U.S. Food and Drug Administration (FDA) on a plan to lift the clinical hold on the Phase 1/2 INGLAXA clinical trial in the United States for 4D-310 for Fabry disease cardiomyopathy. |
| 2023-02-22 | 4D Molecular Therapeutics | Results 4D Molecular Therapeutics Presents Interim Data from 4D-310 INGLAXA Phase 1/2 Clinical Trials & Development Plans for Fabry Disease Cardiomyopathy at WORLDSymposium™ 4dmoleculartherapeutics.com ↗
All three patients with 12 months of follow-up demonstrated improvement on cardiac contractility, exercise capacity and quality of life endpoints. |
| 2023-01-09 | 4D Molecular Therapeutics | Results 4D Molecular Therapeutics Announces Updates on Clinical Pipeline and Additional Preclinical Programs 4dmoleculartherapeutics.com ↗
Reports Program Updates and Interim Clinical Data from Phase 1/2 Fabry Disease Cardiomyopathy Trials; Full Clinical Data to be Reported at WORLDSymposium in February 2023 |
| 2022-02-09 | 4D Molecular Therapeutics | Results 4D Molecular Therapeutics Announces Updated Interim Results from the 4D-310 Phase 1/2 Clinical Trial in Patients with Fabry Disease at the 18th Annual WORLDSymposium 4dmoleculartherapeutics.com ↗
4D Molecular Therapeutics, Inc. (Nasdaq: FDMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced updated interim clinical data from the ongoing Phase 1/2 clinical trial of 4D-310 in patients with Fabry disease at the 18 th Annual WORLD Symposium. |
| 2021-10-25 | 4D Molecular Therapeutics | Results 4D Molecular Therapeutics Reports Interim Results from the 4D-310 Phase 1/2 Clinical Trial in Patients with Fabry Disease and Provides Clinical Data Update from the 4D-110 Phase 1/2 Clinical Trial in Patients with Choroideremia 4dmoleculartherapeutics.com ↗ |
| 2020-08-13 | 4D Molecular Therapeutics | Regulatory 4D Molecular Therapeutics Receives FDA Fast Track Designation for 4D-310 Gene Therapy for Treatment of Fabry Disease 4dmoleculartherapeutics.com ↗ |
| 2019-05-30 | 4D Molecular Therapeutics | Results 4D Molecular Therapeutics Announces Presentation of Preclinical Data at the 6th International Update on Fabry Disease and Provides Clinical Update 4dmoleculartherapeutics.com ↗
Dose dependent GLA expression and function after administration of 4D-310, that incorporates the 4D-C102 vector, in vitro in Fabry patient fibroblasts |
All press releases naming this drug 14 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | DUVALGAGENE OTIPARVOVEC | ChEMBL registry synonym — accepted as the source's own label CHEMBL6068111 ↗ |
| Known as | 4D-310 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05629559 ↗ |
| Known as | 4D-C102 | ChEMBL registry synonym — accepted as the source's own label CHEMBL6068111 ↗ |
| Known as | Duvalgagen otiparvovec | ChEMBL registry synonym — accepted as the source's own label CHEMBL6068111 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “4D-310 is a novel adeno-associated virus (AAV) gene therapy” NCT04519749 ↗ |
| Route | Intravenous | “following a single IV administration” NCT04519749 ↗ |