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Amicus pulls plug on rare skin disease drug after Phase 3 failure

Published on 14/09/17 at 08:33am

New Jersey-based biotech firm Amicus Therapeutics has been forced to terminate its development of its candidate for treating rare skin disease epidermolysis bullosa (EB) after it failed to show any significant efficacy over placebo in Phase 3 trials.

EB is an inherited connective tissue disease which prevents the production of proteins needed to bind layers of skin together, resulting in easily wounded skin and painful blisters. There is currently no approved treatment for the disease, and the only management of the condition is achieved through regular bandaging and cleaning of wounds, which remains burdening and painful process.

In the 139-participant study evaluating the drug’s ability to promote wound closure over three months compared to placebo. It was found that 49% of patients treated achieved target wound closure within the specified time frame – 5% lower than those in the placebo group.

“In seeking to develop novel, high quality therapies for those living with devastating rare diseases we may sometimes fail," commented Amicus CEO John Crowley. "But we would rather be the first to fail, than the last to try.”

Amicus has stated that it has no plans to pursue further development of the drug, but will offer the treatment to the trial’s participants through ongoing extension studies.

News of the failure hit the biotech hard, knocking its shares down by around 15%, though the company has seen its shares double in value since the beginning of the year.

Matt Fellows

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