drugset

Drugs / NIMATPAGENE PARIPARVOVEC

Follow last change Dec 2024 re-read 3 minutes ago

NIMATPAGENE PARIPARVOVEC

also known as Nimatpagen pariparvovec · VTX-801

Gene therapy (AAV / viral vector)

Developed for: Wilson disease
Investigated by: Vivet Therapeutics SAS

Trials 1

today NCT04537377 · Ph 1/2

20222023202420252026

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 1/2	NCT04537377	Sep 2021 → Dec 2024 overdue	Wilson disease	Vivet Therapeutics SAS	Active not recruiting	No outcome recorded

News releases announcing trial results or a regulatory action · 5

Date	Issuer	Release
2024-06-05	Vivet Therapeutics SAS	Results Vivet Therapeutics presents interim data on its Phase 1/2 GATEWAY trial for the Treatment of Wilson Disease at EASL Congress 2024 vivet-therapeutics.com ↗ VTX-801 increased ceruloplasmin ferroxidase activity and improved liver histology
2021-08-12	Vivet Therapeutics SAS	Regulatory VTX‐801 Receives U.S. FDA Fast Track Designation for the Treatment of Wilson Disease vivet-therapeutics.com ↗
2021-08-12	Pfizer	Regulatory VTX-801 Receives U.S. FDA Fast Track Designation for the Treatment of Wilson Disease pfizer.com ↗
2020-11-18	Vivet Therapeutics SAS	Regulatory Vivet and Pfizer Inc. Announce FDA Authorization to Proceed with GATEWAY, the Phase 1/2 Study for VTX-801, Vivet’s Investigational Gene Therapy for WD vivet-therapeutics.com ↗
2017-09-27	Vivet Therapeutics SAS	Regulatory Vivet’s First Gene Therapy Product, VTX 801 for Wilson’s Disease, Receives European and US Orphan Drug Designation vivet-therapeutics.com ↗

All press releases naming this drug 6 releases

Date	Issuer	Release
2024-06-05	Vivet Therapeutics SAS	Vivet Therapeutics presents interim data on its Phase 1/2 GATEWAY trial for the Treatment of Wilson Disease at EASL Congress 2024 vivet-therapeutics.com ↗
2021-08-12	Vivet Therapeutics SAS	VTX‐801 Receives U.S. FDA Fast Track Designation for the Treatment of Wilson Disease vivet-therapeutics.com ↗
2021-08-12	Pfizer	VTX-801 Receives U.S. FDA Fast Track Designation for the Treatment of Wilson Disease pfizer.com ↗
2020-11-18	Vivet Therapeutics SAS	Vivet and Pfizer Inc. Announce FDA Authorization to Proceed with GATEWAY, the Phase 1/2 Study for VTX-801, Vivet’s Investigational Gene Therapy for WD vivet-therapeutics.com ↗
2020-09-15	Vivet Therapeutics SAS	Vivet’s lead program, VTX-801 for Wilson Disease, highlighted during Pfizer’s Investor Day September 15th 2020 vivet-therapeutics.com ↗
2017-09-27	Vivet Therapeutics SAS	Vivet’s First Gene Therapy Product, VTX 801 for Wilson’s Disease, Receives European and US Orphan Drug Designation vivet-therapeutics.com ↗

Evidence & citations 5 cited values

Every value below carries the sentence it was read from. 2 sources stand behind the page.

Field	Value	Cited text
Known as	NIMATPAGENE PARIPARVOVEC	ChEMBL registry synonym — accepted as the source's own label CHEMBL5314853 ↗
Known as	Nimatpagen pariparvovec	ChEMBL registry synonym — accepted as the source's own label CHEMBL5314853 ↗
Known as	VTX-801	ClinicalTrials.gov intervention name — accepted as the source's own label NCT04537377 ↗
Modality	Gene therapy (AAV / viral vector)	“a replication-deficient recombinant adeno-associated viral vector (rAAV)” NCT04537377 ↗
Route	Intravenous	“administered intravenously (IV)” NCT04537377 ↗