Orphan Drug for Rare Skin Disease Fails Clinical Trial

September 15, 2017

Amicus Therapeutics’ proposed treatment for a rare skin disease failed to outperform a placebo in a Phase III clinical trial, the company said in announcing the failure of the developmental program for the medication.

Amicus was testing its topical treatment for epidermolysis bullosa, a genetic disorder that leads to severe skin blistering and open wounds, often beginning at birth. There are no approved therapies to treat the condition.

The medicine had received designations from the FDA for a rare pediatric disease and as an orphan drug and breakthrough therapy.

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