Contribute Try STAT+ Today

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.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

GET STARTED

What is it?

STAT+ is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.

What's included?

  • Daily reporting and analysis
  • The most comprehensive industry coverage from a powerhouse team of reporters
  • Subscriber-only newsletters
  • Daily newsletters to brief you on the most important industry news of the day
  • STAT+ Conversations
  • Weekly opportunities to engage with our reporters and leading industry experts in live video conversations
  • Exclusive industry events
  • Premium access to subscriber-only networking events around the country
  • The best reporters in the industry
  • The most trusted and well-connected newsroom in the health care industry
  • And much more
  • Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr.