VTX-801
An investigational treatment for Wilson Disease.
Drug facts
The same compound appears under different names depending on the context. Here is how to identify VTX-801 wherever you encounter it, plus the key facts at a glance.
- Generic name
- VTX-801
- Development code
- VTX-801
- Drug class
- AAV gene therapy (ATP7B)
- Manufacturer
- Vivet Therapeutics
A gene therapy for Wilson disease from Vivet Therapeutics that is no longer in development.
According to the Wilson Disease Association, Vivet ended the Phase 1/2 GATEWAY trial in October 2024 after treating 4 patients at 2 doses, finding it safe but not effective enough; those patients remain in follow-up.
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Where VTX-801 fits
No longer in development. Other gene-based approaches are still being tested, including Ultragenyx's UX701 and Prime Medicine's PM577a, so ask your hepatologist about current Wilson disease trials.
How VTX-801 works
Investigational AAV-based gene therapy that delivers a functional copy of the ATP7B gene to liver cells, aiming to restore the body's ability to excrete excess copper through bile and provide a long-lasting alternative to lifelong chelation or zinc therapy
Clinical trial results
The Phase 1/2 GATEWAY trial (a first-in-human, open-label, dose-escalation, 5-year follow-up study) is evaluating safety, pharmacodynamics, and efficacy of a single VTX-801 infusion. Cohort 1 dosing showed encouraging safety, tolerability, and early pharmacodynamic effects. A first patient in the second dose group was then dosed. Interim data presented at EASL 2024 showed increases from baseline in ceruloplasmin activity in 2 patients in the first dose group.
Vivet ended the trial in October 2024 after 4 patients at 2 doses, according to the Wilson Disease Association, and ClinicalTrials.gov lists it as active but not recruiting while treated patients are followed.
Main registered trial: NCT04537377 on ClinicalTrials.gov. Check it for the current status, sites and contacts before asking about enrollment.
Side effects and safety
Only a small number of patients have received VTX-801 so far, so its safety picture is still early. In the first dose group of the GATEWAY trial, the therapy was generally well tolerated, and Vivet reported no serious adverse events. One patient had a reaction during the infusion itself, which was managed with medication, and both patients went home the day after treatment as planned.
Some patients had temporary rises in liver enzymes, an expected and watched-for effect with gene therapies delivered to the liver, and these were manageable[1]. The Wilson Disease Association reports that the therapy was safe at both doses.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Development history
Developed by Vivet Therapeutics, a Paris-based company with a research site in Pamplona, Spain. Pfizer invested in Vivet in 2019. The FDA granted VTX-801 Fast Track designation for Wilson disease in August 2021, and the GATEWAY trial followed. Vivet ended the program in October 2024 after 4 patients, finding the therapy safe but not effective enough (Wilson Disease Association).
Explore Wilson Disease trials
Other Wilson Disease treatments
Common questions about VTX-801
▸What is VTX-801?
A gene therapy for Wilson disease from Vivet Therapeutics that is no longer in development. According to the Wilson Disease Association, Vivet ended the Phase 1/2 GATEWAY trial in October 2024 after treating 4 patients at 2 doses, finding it safe but not effective enough; those patients remain in follow-up.
▸What is the mechanism of action of VTX-801?
Investigational AAV-based gene therapy that delivers a functional copy of the ATP7B gene to liver cells, aiming to restore the body's ability to excrete excess copper through bile and provide a long-lasting alternative to lifelong chelation or zinc therapy
▸What are the side effects of VTX-801?
Only a small number of patients have received VTX-801 so far, so its safety picture is still early. In the first dose group of the GATEWAY trial, the therapy was generally well tolerated, and Vivet reported no serious adverse events. One patient had a reaction during the infusion itself, which was managed with medication, and both patients went home the day after treatment as planned. Some patients had temporary rises in liver enzymes, an expected and watched-for effect with gene therapies delivered to the liver, and these were manageable[1]. The Wilson Disease Association reports that the therapy was safe at both doses.
▸Is VTX-801 FDA approved?
VTX-801 is currently in discontinued clinical trials for Wilson Disease. It has not yet received FDA approval.
▸What is VTX-801?
VTX-801 was a one-time gene therapy infusion for Wilson disease from Vivet Therapeutics, meant to give liver cells a working ATP7B gene. Vivet ended the program in October 2024 after the GATEWAY trial treated 4 patients, finding it safe but not effective enough. [3][4]
▸Is VTX-801 FDA approved?
No. VTX-801 was never approved. Vivet ended the GATEWAY trial in October 2024 and has not announced further development, according to the Wilson Disease Association. The FDA had granted it Fast Track designation in August 2021, which speeds development and review but is not an approval. [2][3]
▸What has the VTX-801 GATEWAY trial shown?
GATEWAY is a first-in-human, open-label, dose-escalation study of a single VTX-801 infusion with 5 years of follow-up. Interim data presented at EASL 2024 showed increases in ceruloplasmin activity in 2 patients in the first dose group, and a first patient in the second dose group was then dosed. Vivet then ended the trial in October 2024 after 4 patients, finding the therapy safe but not effective enough; treated patients remain in follow-up. [1][3][4]
▸What are the side effects of VTX-801?
Only a small number of patients have received VTX-801, so its safety picture is still early. In the first dose group of GATEWAY, the therapy was generally well tolerated, and Vivet reported no serious adverse events; 1 patient had a reaction during the infusion that was managed with medication, and some patients had temporary, manageable rises in liver enzymes. The Wilson Disease Association reports that the therapy was safe at both doses. [1][3]
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- Vivet Therapeutics. Vivet Therapeutics presents interim data on its Phase 1/2 GATEWAY trial for Wilson disease at EASL Congress 2024. https://www.vivet-therapeutics.com/vivet-therapeutics-presents-interim-data-on-its-phase-1-2-gateway-trial-for-the-treatment-of-wilson-disease-at-easl-congress-2024/
- Pfizer. VTX-801 Receives U.S. FDA Fast Track Designation for the Treatment of Wilson Disease. https://www.pfizer.com/news/press-release/press-release-detail/vtx-801-receives-us-fda-fast-track-designation-treatment
- Wilson Disease Association. Genetic therapies for Wilson disease. https://wilsondisease.org/living-with-wilson-disease/treatment/genetic-therapies/
- ClinicalTrials.gov. A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease (GATEWAY). https://clinicaltrials.gov/study/NCT04537377