STAT+: Amicus drug for Pompe disease falls short in key clinical trial, but company will push for approval
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.
Continue to STAT+ to read the full story…
