Pfizer Vyndaqel Advisory Committee Pits Orphan Flexibility Vs. Data Shortcomings
This article was originally published in The Pink Sheet Daily
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.
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Rare disease drug development has ballooned in just three years, powered by the success of independent biotechs like Alexion, Big Pharma’s entrée into the field, and pressure from regulators and payors that is dis-incentivizing development of traditional primary care drugs. The positive momentum has intensified dealmaking in the orphan drug space and is giving investors confidence to back rare disease-focused start-ups. Some have coined the resulting movement the “orphan drug bubble,” but interest isn’t likely to deflate soon – at least not as long as Big Pharma continues to invest in the area and industry is able to sustain a favorable reimbursement climate for ultra-premium-priced drugs.
When Is A Single Pivotal Trial “Robust” Enough? FDA’s Katz Offers Examples That Could Spell Trouble For Pfizer’s Vyndaqel
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.
Pfizer gained the therapy to treat a rare genetic neurologic disorder in its 2010 acquisition of folded protein company FoldRx.