Amicus Therapeutics drug for kids born with rare skin disease fails late-stage trials

By Business Leaders FOXBusiness

A father's fight to find a cure for his children's rare disease

Amicus Therapeutics CEO John Crowley on how his fight to find a cure for his children's rare disease, Pompe, has driven his work in the pharmaceutical industry.

Amicus Therapeutics (FOLD) CEO John Crowley told FOX Business on Wednesday the company’s drug to treat a rare wound disease failed late-stage clinical trials.

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“This was one product for just a devastating rare disease, as you said called EB [Epidermolysis Bullosa],” he told Maria Bartiromo during an exclusive interview on Mornings with Maria.

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“You may have heard these children referred to as “butterfly children.” Their skin is so fragile; imagine living with a rare disease where your skin literally will fall off, develop open wounds and sores — just devastating. So we had acquired a company, developed a medicine and it didn’t meet its primary goals in its phase 3.”

Despite the setback, Crowley said the company has never been in a better position to invest in its pipeline.

“We have great science, a great portfolio, a very strong balance sheet. This has been a tremendous year for Amicus. We have more milestones ahead of us. We have, in the next couple of weeks, complete data in our phase 2 study in Pompe, by the end of the year, for our February drug that’s approved in Europe. We’ll be filing here in the United States, [and] we just filed for approval in Japan.”

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