Alnylam's Amvuttra Enters ATTR Cardiomyopathy Market with Premium Pricing Strategy

Alnylam Pharmaceuticals has secured FDA approval for Amvuttra in the treatment of transthyretin amyloidosis (ATTR) cardiomyopathy, marking a significant expansion of the drug's indications. The company's pricing strategy and market positioning have sparked discussions among industry analysts and investors.

Amvuttra's Pricing and Market Entry

Alnylam has set Amvuttra's annual list price at approximately $476,000, nearly double the cost of existing treatments from Pfizer and BridgeBio. This pricing decision comes as Amvuttra enters a market already occupied by Pfizer's tafamidis and BridgeBio's Attruby, both oral medications priced around $250,000 and $244,000 per year, respectively.

Tolga Tanguler, Alnylam's Chief Commercial Officer, defended the pricing strategy, stating that Amvuttra "offers a compelling and highly differentiated value for patients and physicians, as well as payers." The company cites several factors to justify the premium, including:

• Amvuttra's unique mechanism of action, which "silences" the misfolded protein implicated in ATTR rather than stabilizing it
• Approval for both forms of ATTR (polyneuropathy and cardiomyopathy)
• Demonstrated benefits in patients with less advanced disease and those already on other treatments
• A quarterly injection regimen that may improve treatment adherence compared to daily oral medications

Market Dynamics and Competitive Landscape

The ATTR cardiomyopathy market is expected to grow significantly, with Alnylam estimating that the majority of an estimated 150,000 patients in the U.S. remain undiagnosed. Pfizer's tafamidis generated over $5 billion in sales last year, while BridgeBio's Attruby has shown promising early prescription numbers.

Amvuttra's entry into this competitive landscape has been met with mixed reactions from analysts:

• Stifel analyst Paul Matteis views Alnylam's pricing strategy as sensible, noting that Amvuttra's administration through Medicare Part B could provide advantages in terms of reimbursement and patient access.
• Leerink Partners analyst Mani Foroohar cautioned that the high price could lead to "significant payer headwinds to commercial uptake" and suggested that Wall Street projections for Amvuttra's market share may be overly optimistic.

Clinical Profile and Regulatory Details

The FDA approval of Amvuttra for ATTR cardiomyopathy is based on its ability to reduce hospitalizations or death resulting from heart complications. The drug's label also includes mention of urgent care visits for heart failure, which Alnylam's Chief Medical Officer Pushkal Garg highlighted as "unique" to their product.

While direct comparisons between Amvuttra and its competitors are limited due to the lack of head-to-head trials, Alnylam emphasized the drug's performance in studies involving patients with less advanced disease and those already receiving other treatments.

Alnylam projects $1.6 billion to $1.7 billion in sales for its ATTR business in 2025, which includes Amvuttra and an older drug for polyneuropathy. The company expects initial uptake to be gradual, with acceleration anticipated later this year.