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When Is A Single Pivotal Trial “Robust” Enough? FDA’s Katz Offers Examples That Could Spell Trouble For Pfizer’s Vyndaqel

 
• By Martin Berman-Gorvine

The single pivotal trial of Pfizer’s Vyndaqel couldn’t offer p-values of less than 0.01, nor substantial evidence from multiple sites, which could spell trouble for the new drug application.

When FDA receives a new drug application with a single pivotal trial plus confirmatory evidence offered in support of accelerated approval, for example Pfizer Inc.’s orphan drug Vyndaqel (tafamidis meglumine) for familial amyloid polyneuropathy, the agency sets a high standard for statistical significance, such as a p-value of less than 0.01, an FDA official said.

While stressing that there is not a hard-and-fast rule, Russell Katz, director of FDA’s Division of Neurology Products, told the...

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US FDA’s CRL Release More Incremental Than ‘Radical’ Transparency

 
• By Bridget Silverman

The transparency initiative collects CRLs already made public in approval packages on its drugs@fda site to post on the openFDA public portal.

US FDA Asking Questions About Commissioner’s Voucher Program With Details Scarce

 
• By Derrick Gingery

FDA staff are trying to plan for program implementation in the absence of critical eligibility and other details, although guidance may be coming soon.

Senate Appropriators Give US FDA More Non-User Fee Funds Than House

 
• By Sarah Karlin-Smith

US FDA would receive more funding for fiscal year 2026 in the Senate appropriations bill than its House counterpart, including $10m for unannounced foreign inspections.

Animal Testing: US And EU Regulatory Support Grows For Alternatives, But Validation A Hurdle

 
• By Eliza Slawther

In this first of a two-part series of articles, US Pharmacopeia CEO Ronald T. Piervincenzi tells the Pink Sheet how the use of alternatives to animal testing in drug development is gaining traction and discusses the challenges that exist.

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Senate Panel Approves US CDC Director Candidate Monarez With Measles Outbreak Growing

 
• By Cathy Kelly

The Senate Health, Education, Labor and Pensions Committee voted to advance Monarez's nomination to lead the CDC as the agency announced the largest US measles outbreak in 22 years.

US FDA Advisory Committee Supports Vaccine Safety, Scrutinizes AE Reporting Quality

 
• By Sue Sutter

The Pediatric Advisory Committee supported routine safety monitoring for three vaccines and 21 drugs, but the panel’s consumer representative said adverse event reporting needs improvement and FDA literature reviews should include lawsuits as well as social media.

US FDA Will Not Pull Teva And MSN Hetlioz ANDAs, Rejects Vanda Citizen Petitions

 
• By Dean Rudge

The FDA concluded that generic tasimelteon products held by Teva and MSN Labs meet all statutory bioequivalence requirements and there was no “mistake” or untrue statement in the original ANDA approvals.