As Alnylam Pharmaceuticals approaches its highly anticipated March 23 FDA decision date for a potential expansion of Amvuttra to treat patients in an increasingly competitive indication—transthyretin amyloid cardiomyopathy (ATTR-CM)—the company is emphasizing market dynamics.
“The category is rapidly growing and it’s been largely underserved,” Tolga Tanguler, Alnylam’s chief commercial officer said during a conference call Thursday.” This will be a market-growth story.”
After Alnylam divulged last month at the J.P. Morgan Healthcare Conference that it expects Onpattro and Amvuttra to generate between $1.6 billion and $1.7 billion in 2025, there was little suspense left in Thursday’s fourth-quarter earnings presentation about the company’s expectation for its potential launch into the new indication.
The projection compares to the ATTR franchise's combined 2024 sales of $1.23 billion, including $970 million for Amvuttra, which nearly achieved blockbuster status in just its second full year on the market.
Both drugs are approved for polyneuropathy caused by hereditary transthyretin amyloidosis (hATTR-PN), with Onpattro’s FDA nod coming in 2018.
With sales of Onpattro and Amvuttra increasing by 33% year over year and 30% in the fourth quarter of 2024, there is already lots of momentum without the new indication. While entrance into the ATTR-CM market may not contribute much to the top line in 2025, the Massachusetts biotech is optimistic about its long-term potential.
“It is a category ripe for an entirely new treatment option. In this context, we have the potential to introduce a disruptive treatment,” Tanguler said. “Assuming approval, Amvuttra would be the first and only RNAi approved for both ATTR-CM and hATTR-PN.”
In ATTR-CM, BridgeBio has the same disruptive expectation for its transthyretin stabilizer Attruby, which was approved by the FDA three months ago and is set to challenge Pfizer’s blockbuster Vyndaqel franchise.
In the fourth quarter, Alnylam reported net product revenue of $451 million, which was a 30% increase year over year. For 2025, Alnylam is projecting product revenue of between $2.05 billion to $2.25 billion, which would be a 31% increase at midpoint.