The FDA Fast Tracks Therapy for Rare Disease Causing Copper Accumulation

Wilson's Disease

The FDA is speeding up the process for regulatory approval of WTX101, a therapy for Wilson’s disease that could significantly improve the standard of care. 

Wilson Therapeutics, in Stockholm, has convinced the FDA to include its drug WTX101 (bis-choline tetrathiomolybdate) in its Fast Track program. 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.

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. Wilson Therapeutics estimates that current treatments are not effective in 45% of patients and can have severe side effects such as lupus or myasthenia. WTX101 binds copper and helps the body excrete it, which could be a safer and more effective treatment alternative.

The FDA has granted WTX101 Fast Track Designation based on data from a Phase II trial. In it, the drug was able to reduce copper levels in patients with Wilson’s disease by 72%. The good news from the FDA comes just a week after Wilson Therapeutics raised €24.4M on Nasdaq Stockholm that will be dedicated to fund late-stage development of WTX101 and to prepare for its commercialization.


Image via Nebuto /Shutterstock

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