VTX-801
An investigational treatment for Wilson Disease.
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 (in partnership with Pfizer)
An investigational AAV-based gene therapy for Wilson disease developed by Vivet Therapeutics in partnership with Pfizer. VTX-801 received FDA Fast Track designation in 2021 and is being studied in the Phase 1/2 GATEWAY trial. Interim data presented at EASL 2024 showed restored ceruloplasmin ferroxidase activity, improved liver histology, and no serious adverse events.
Where VTX-801 fits
Investigational, not currently approved. If GATEWAY results continue to support safety and efficacy, VTX-801 would be one of the first gene therapies for Wilson disease and a potential alternative to lifelong chelation or zinc maintenance. GATEWAY is not recruiting, so patients should ask their hepatologist about other Wilson disease trials listed on ClinicalTrials.gov.
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
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 with no unexpected serious side effects. 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 full safety profile will take shape as the trial moves through higher dose groups and longer follow-up.
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.
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. The first patient in Cohort 2 was dosed in April 2024. Interim data presented at EASL 2024 showed increased ceruloplasmin ferroxidase activity and improved liver histology. ClinicalTrials.gov now lists GATEWAY as active but not recruiting, with 4 patients enrolled.
Development history
VTX-801 originated at Vivet Therapeutics in Spain. Pfizer secured an exclusive option to acquire Vivet in 2019 and took a 15% equity stake. The FDA granted VTX-801 Fast Track designation for Wilson disease in August 2021. The GATEWAY clinical trial began shortly thereafter. It is no longer recruiting, and treated patients remain in follow-up.
Explore Wilson Disease trials
Other Wilson Disease treatments
Common questions about VTX-801
▸What is VTX-801?
An investigational AAV-based gene therapy for Wilson disease developed by Vivet Therapeutics in partnership with Pfizer. VTX-801 received FDA Fast Track designation in 2021 and is being studied in the Phase 1/2 GATEWAY trial. Interim data presented at EASL 2024 showed restored ceruloplasmin ferroxidase activity, improved liver histology, and no serious adverse events.
▸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 with no unexpected serious side effects. 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 full safety profile will take shape as the trial moves through higher dose groups and longer follow-up.
▸Is VTX-801 FDA approved?
VTX-801 is currently in phase 2 clinical trials for Wilson Disease. It has not yet received FDA approval.
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
- ClinicalTrials.gov. A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease (GATEWAY). https://clinicaltrials.gov/study/NCT04537377