Myozyme for Pompe disease, now in its final phase of clinical trials, is potentially next.

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One said that the agency would not approve Lumizyme, to treat a rare genetic disorder called Pompe disease, before a deadline of Feb. 28, as expected.

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Pharming, a Dutch biotechnology firm, recently began clinical trials for alpha-glucosidase, a product designed to treat Pompe's disease, a metabolic disorder causing fatal degeneration of muscle.

ECONOMIST: Rare diseases: Safety in numbers | The

The drug started as an attempt to simply expand the production of one of these treatments, Myozyme, which is used to replace a missing enzyme for patients with Pompe disease.

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An illustration earlier this year was the delay in FDA approval of a biological used to treat Pompe disease--a debilitating inherited disorder--that was to be manufactured in a new, larger-scale manufacturing facility.

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