Wilson’s disease Drug Granted Fast Track Designation by the FDA

Wilson’s disease Drug Granted Fast Track Designation by the FDA

Wilson’s disease is a rare genetic disorder that affects copper metabolism, leading the metal to accumulate in the body and cause severe damage, especially to the liver and the nervous system. Current treatments are not effective in 45% of patients and can have severe side effects such as lupus or myasthenia.

Stockholm-based biotech Wilson Therapeutics’ in-trial drug, WTX101, is a drug with active ingredient bis-cholin

e tetrathiomolybdate, a molecule that binds copper in a way that helps the body excrete it. It is a first-in-class copper-protein-binding agent with a unique mechanism of action, under investigation as a novel therapy for Wilson Disease. WTX101 has successfully completed a Phase II clinical study, where it showed to reduce copper concentration in patients with Wilson’s disease by 72%.

The firm has now announced that the U.S. Food and Drug Administration (FDA) has granted WTX101 Fast Track designation for the treatment of Wilson Disease. The FDA’s Fast Track program is designed to facilitate the development, and expedite the review of novel therapies to treat serious conditions and fill an unmet medical need.

This move will speed up the approval of the drug, though still subject to obtaining positive results in an upcoming Phase III trial planned to start in early 2018.

The Fast Track Designation is supported by data from the 24-week open label multicenter 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 stabilize 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 enroll the first patient in the Phase 3 FOCuS trial in early 2018.

“We are extremely pleased to receive Fast Track designation from the FDA, which supports our view that WTX101 can address significant unmet medical needs in this rare and debilitating disease. The Fast Track designation also provides a number of regulatory advantages, including additional access to the FDA which can speed up the future review of WTX101 and help bring this drug to patients as quickly as possible,” commented Wilson Therapeutics CEO Jonas Hansson.