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Pfizer Vyndaqel Advisory Committee Pits Orphan Flexibility Vs. Data Shortcomings

 
• By Martin Berman-Gorvine

FDA finds much to question in a single pivotal trial of Pfizer’s orphan drug Vyndaqel (tafamidis meglumine) for familial amyloid polyneuropathy, but the ultra-orphan nature of the disease may outweigh those concerns.

Pfizer Inc. must be hoping to appeal to FDA’s flexibility on orphan drug reviews as the advisory panel tasked with reviewing Vyndaqel (tafamidis meglumine) weighs a dispute between agency and sponsor over the statistical reading of the pivotal trial results, and the agency notes ominously that the bulk of the efficacy seems to come from a single, foreign site.

Most of FDA’s approval decisions for orphan products use unconventional efficacy standards, according to a report released last year by...

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More from United States

Real-Time Release Of CRLs Is US FDA’s Goal, Agency Leaders Affirm

 
• By Michael McCaughan

The US FDA says its release of complete response letters for products subsequently approved by the agency is just a first step toward releasing rejection letters when they are issued, assuming legal issues can be resolved.

No Surprise Here: Foreign Drug Facilities Should Expect Surprise Inspections, US FDA Says

 
• By Sue Sutter

The FDA's recent announcement of plans to expand unannounced inspections at ex-US facilities should put firms on advance notice that their next foreign facility inspection may not be preannounced, Ivy Sweeney, acting head of the agency's drug inspections office said.

Base Benefit-Risk Decisions On Data, Not Opinion And Passion, CDER’s Tidmarsh Says

 
• By Sue Sutter

New US FDA drugs center director George Tidmarsh’s data-centric comments at a Reagan-Udall Foundation meeting on unapproved fluoride products could suggest patients’ and caregivers’ experiences will carry less weight in benefit-risk decisions under his leadership.

Pink Sheet Podcast: FDA’s New Top Drug Regulator, Sarepta’s Options After Elevidys Stand-Off

 
• By Derrick Gingery, Sarah Karlin-Smith, and Nielsen Hobbs

Pink Sheet reporter and editors discuss the potential impact George Tidmarsh could have as director of the US FDA Center for Drug Evaluation and Research and the issues threatening the future of Sarepta’s gene therapy Elevidys.

More from North America

Merck’s Keytruda Gains Medicare Negotiation Reprieve With Expanded Orphan Exemption

 
• By Cathy Kelly

Bristol Myers Squibb’s Opdivo and Merck's Welireg also could benefit from the delay in Medicare price negotiation process eligibility.

US FDA’s Biologics Center Departures Grew Before More Than 100 RIFs Exited

 
• By Derrick Gingery

The Center for Biologics Evaluation and Research saw departures far outnumber new hires in the second and third quarters of FY 2025, before more than 100 people left July 14 after the reduction-in-force was finalized.

US FDA Cancer Reviewers Heading For The Exits, Potentially Impacting Review Timelines

 
• By Sarah Karlin-Smith

US FDA's cancer drugs division is feeling the effect of drug reviewer departures, despite efforts to keep them at the agency.