1. Pink Sheet
  2. >>Product Reviews
  3. >>Approvals

FDA Reversal Gives Amicus Renewed Hope For US Oral Fabry Launch

 
• By Jessica Merrill

Amicus plans to file migalastat for Fabry disease in the US in the fourth quarter, after FDA gave the greenlight, reversing a decision last year to require an additional Phase III study. Additional information includes European submission data and post-launch RWE.

pills-tablets-glass-container_69512212_1200

It's not often that FDA backtracks on a decision, but in a surprising turn of events, the agency has reversed a decision on Amicus Therapeutics Inc.'s oral therapy for Fabry disease, migalastat, agreeing the company can submit an NDA for accelerated review based on existing data.

The company announced July 11 that it now plans to file an NDA for migalastat in the fourth quarter, following a series of discussions and written communications with the agency, which could

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from Approvals

More from Product Reviews

Recent and Upcoming FDA Advisory Committee Meetings

 
• By Sue Sutter

Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.

US FDA Advisory Committee Misperceptions Abound … At HHS

 
• By Michael McCaughan

HHS Secretary Robert F. Kennedy Jr. regularly decries the “conflicts of interest” he believes abound in advisory committees, but his concerns, as well as a recent “policy directive” eliminating industry representatives, seem driven by a misunderstanding of the panels’ jobs.

US FDA Layoffs Hit Conflict-Of-Interest Screening For ODAC Meeting

 
• By Sue Sutter and Derrick Gingery

Screening for conflicts and finding the right expertise for a two-day Oncologic Drugs Advisory Committee meeting on four products has been complicated by the Trump Administration’s initial communications freeze and subsequent layoffs in CDER.