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US FDA’s Opaque Galafold Review: A Rare Misstep In The Trump Era

 
• By Michael McCaughan

When FDA reversed course and allowed Amicus to file for Accelerated Approval of migalastat, plenty of people suspected politics were at play. The agency's handling of the review won’t do anything to allay those suspicions.

Galafolds' heavily redacted cardio-renal consult review
What's the whole story of the Galafolds review?

US FDA Commissioner Scott Gottlieb has earned high marks for running the agency independently and credibly in the era of President Donald Trump.

Against that backdrop, FDA’s handling of Amicus Therapeutics Inc

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US FDA Vaccine Framework Coming In Weeks, But Details Vague

 
• By Sarah Karlin-Smith

New CBER Director Prasad will "unleash a massive" framework on vaccines, FDA Commissioner Marty Makary said 15 May, raising questions about the impact on products nearing approval.

HHS Secretary Kennedy Says Trump Will Make Final Decision On Mifepristone Policy

 
• By Derrick Gingery

With an FDA review ongoing, Kennedy’s admission, made during a Senate hearing on the Health and Human Services Department’s budget request, suggests political officials could supersede scientific decisions.

US FDA Waiting On Guidance Agenda Future As HHS Seeks Regulation Cuts

 
• By Derrick Gingery

The FDA continues to wait for a decision on whether its priority guidances will be published as HHS asks the public for potential regulation cuts to fulfill President Trump’s executive order.

US FDA Rare Disease Case Studies Provide Development Models For Sponsors

 
• By Laura Helbling

Sanofi’s Xenpozyme and Sentynyl’s Nulibry are the first two case studies the FDA is using to continue educating rare disease sponsors on best practices.

More from Pathways & Standards

US HHS Deputy Nominee Talks AI, Not FDA, In Confirmation Hearing

 
• By Ramsey Baghdadi

Silicon Valley investor Jim O’Neill’s Senate confirmation hearings showcased the unique background of someone with government and tech experience and avoided discussions of his past statements about lowering the bar for regulatory approval.

US FDA Waiting On Guidance Agenda Future As HHS Seeks Regulation Cuts

 
• By Derrick Gingery

The FDA continues to wait for a decision on whether its priority guidances will be published as HHS asks the public for potential regulation cuts to fulfill President Trump’s executive order.

US FDA Rare Disease Case Studies Provide Development Models For Sponsors

 
• By Laura Helbling

Sanofi’s Xenpozyme and Sentynyl’s Nulibry are the first two case studies the FDA is using to continue educating rare disease sponsors on best practices.