Amicus's fat accumulation drug effective in second late-stage study

[August 20, 2014]  (Reuters) - Amicus Therapeutics Inc said its experimental drug was as effective as enzyme replacement therapies (ERTs) after 18 months in patients with an inherited disorder that leads to the abnormal build-up of fat.

The company's shares soared 60 percent in premarket trading.

Amicus said in April that the drug, migalastat HCl, significantly reduced the abnormal accumulation of fat, compared with a placebo, related to a rare genetic disorder that could lead to heart attack, stroke and kidney failure.

The second late-stage trial was testing the drug against standard-of-care ERTs in 60 patients with a form of Fabry disease, Amicus said on Wednesday.

Migalastat had a comparable effect to ERT on patients' kidney function - the main goal of the study, the company said.

(Reporting by Natalie Grover in Bangalore; Editing by Savio D'Souza)

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