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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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