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STAT+: Amicus drug for Pompe disease falls short in key clinical trial, but company will push for approval

This post originally appeared on StatNews.

Amicus Therapeutics said Thursday that its drug for patients with Pompe disease, a rare genetic condition, failed to demonstrate superiority over the current standard treatment in a large clinical trial.

The company, however, believes it was close enough to achieving a positive outcome that its results, combined with other data showing patients with Pompe benefit, could still lead to regulatory approvals. It plans to complete a submission to the Food and Drug Administration in the second quarter, with filings elsewhere in the world to follow.

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This post originally appeared on StatNews.

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