Posted by Michael Wonder on 17 Dec 2017
WTX101 granted fast track designation by the U.S. FDA for the treatment of Wilson disease
14 December 2017 - Wilson Therapeutics today announced that the U.S. FDA has granted WTX101 fast track designation for the treatment of Wilson disease.
WTX101 (bis-choline tetrathiomolybdate) is a first-in-class copper-protein-binding agent with a unique mechanism of action, under investigation as a novel therapy for Wilson disease.
The fast track Designation is supported by data from the 24-week open label multi-center Phase 2 trial in patients with Wilson Disease (WTX101-201), which was conducted by Wilson Therapeutics. The study showed that once-daily dosing of WTX101 has the potential to rapidly lower and control free copper, improve or stabilise neurological and liver status and improve patient-reported disability. Furthermore, no cases of initial drug-induced neurological worsening upon treatment initiation have been observed to date. An open-label extension study is ongoing, and the company expects to enrol the first patient in the Phase 3 FOCuS trial in early 2018.
