10. Vyndaqel


Active ingredient: tafamidis meglumine 
Disease: transthyretin-mediated amyloidosis cardiomyopathy
Peak sales estimate: $1.5 billion 
Approved: May 3, 2019 
Company: Pfizer 

The scoop: Alnylam and Ionis may have been first and second to market with a drug for the rare disease transthyretin-mediated amyloidosis (ATTR), but Pfizer’s Vyndaqel may yet carve itself a niche. For starters, it’s approved for ATTR cardiomyopathy, a variant of the disease that affects the heart, rather than the neurodegenerative variant for which Alnylam’s Onpattro and Ionis’ Tegsedi are approved. That said, the drugs’ paths are likely to cross in the future, as Alnylam is pushing toward an approval in ATTR cardiomyopathy and Pfizer, despite an FDA rejection years ago, is hoping to move Vyndaqel into ATTR polyneuropathy. If and when that happens, Vyndaqel may still win—it is a pill, rather than an injection like Onpattro and Tegsedi, and it has a price tag of $225,000 per year compared to $450,000 per year for the other two. And Vyndaqel has hit the ground running. Its launch blew past expectations, raking in $79 million in U.S. third-quarter sales, well above analysts’ expectations of $21 million. — Amirah Al Idrus

10. Vyndaqel

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