news

  • 14 December 2017
    WTX101 granted Fast Track designation by The U.S. FDA for the treatment of Wilson Disease

    Wilson Therapeutics AB (publ), today 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. 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.

    “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.

    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.