dm+d

Unassigned

New Medicines

Wilson's disease

Information

New molecular entity
Vivet Therapeutics
Vivet Therapeutics

Development and Regulatory status

Phase II Clinical Trials
None
Phase II Clinical Trials
Yes
Yes
Aug 21FDA grants fast track designation to VTX 801 for hepatolenticular degeneration (Wilson ´s Disease) [3].
Sep 17Granted orphan drug designation for the treatment of Wilson ’s disease in the EU & US [3].

Category

An in vivo gene therapy using an adeno-associated virus (AAV) containing a gene coding for a functional mini-ATP7B copper transporter, that is intended to restore copper metabolism through liver cell targeting.
Wilsons disease is inherited as an autosomal recessive trait. It is a rare condition and can often be difficult to diagnose. It affects about 1 in 30,000 individuals [1].
Wilson's disease
Intravenous

Trial or other data

Apr 21PI/II GATEWAY trial to assess, for up to 5 years, the safety, tolerability and pharmacological activity of a single ascending doses of VTX 801 administered intravenously to adult patients with Wilson ´s disease starts (VTX-801_CLN_001; NCT04537377). The non-randomised, open label, adaptive design, single dose-escalation study intends to enrol 16 participants in the US and UK (at the Royal Surrey Hospital). VTX-801 will be administered as a single dose intravenous (IV) administration per patient, at up to 3 different dose levels. Primary outcome is safety and tolerability profile; collection of these data is due to complete Jul 23 [2,4].