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STAT+: Alnylam, once in the hunt to be a $100 billion biotech, needs to solve its heart drug problem

By · Via STAT Health · Published by Everything Blog

In short

The biotechnology firm previously reached a $60 billion valuation and was on track to rival the scale of Vertex Pharmaceuticals. Much of that historical ascent relied on strong commercial sales of Amvuttra, an established treatment for cardiomyopathy. However, mounting concerns tied to the heart medication now pose a significant hurdle to achieving the targeted $100 billion benchmark.

Key points

  • Emerging complications with its heart medication are currently hindering Alnylam's pursui…: Emerging complications with its heart medication are currently hindering Alnylam's pursuit of a twelve-figure valuation.
  • Strong sales of the blockbuster drug Amvuttra remain the primary growth engine for the bi…: Strong sales of the blockbuster drug Amvuttra remain the primary growth engine for the biotechnology firm.
  • Industry comparisons to peer company Vertex Pharmaceuticals have been disrupted due to on…: Industry comparisons to peer company Vertex Pharmaceuticals have been disrupted due to ongoing drug challenges.

Sources checked by our analysis pipeline: 1

This story first appeared in Adam’s Biotech Scorecard, a STAT newsletter. Sign up here to get it delivered to your inbox. And subscribe to STAT+ to get an unabridged version, with exclusive content.

Accountability check: Let’s talk about my “Alnylam is the next Vertex” take from the Aug. 7, 2025, edition of this newsletter.

Alnylam and Vertex are not direct competitors, but I was intrigued by the possibility that Alnylam’s rapidly accelerating growth — fueled by sales of its blockbuster cardiomyopathy treatment Amvuttra — could push its $60 billion market value equal to or higher than Vertex’s $100 billion market cap in the near future.

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