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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.


“Oftentimes, people try to put their best face on data. Here, it’s really easy. We know this drug is having a profound effect on these patients,” Amicus CEO John Crowley said in an interview with STAT. “We have a clear path to approval and we believe this has the potential to become the new standard of care.”

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