Can Alnylam's Broader Portfolio Ease Its Dependence on Amvuttra?

Alnylam Pharmaceuticals’ ALNY primary top-line driver is its newest drug, Amvuttra (vutrisiran), which is approved in the United States and the EU for treating the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis and ATTR amyloidosis with cardiomyopathy (ATTR-CM). The drug’s phenomenal uptake has been driven by new patients starting treatment as well as several patients switching from Onpattro (patisiran), ALNY’s first FDA-approved drug for hATTR amyloidosis.

Alnylam also markets several other products across rare disease and cardiovascular markets, providing the company with incremental revenues that add to the top line.

Givlaari (givosiran) is approved in both the United States and the EU for treating adults with acute hepatic porphyria, with the EU also approving the drug for use in adolescents. Strong uptake has made Givlaari a meaningful revenue driver, and Alnylam is actively working on regulatory submissions in additional regions through 2025 and beyond to widen its global presence.

Similarly, Oxlumo (lumasiran) injection was initially approved in the United States and the EU for the treatment of primary hyperoxaluria type 1, to lower urinary oxalate levels in pediatric and adult patients. Later, the drug’s label was expanded to include lowering urinary and plasma oxalate levels. This expansion, coupled with pending regulatory filings in additional territories, strengthens its potential for international growth.

Alnylam also markets a fifth drug, Leqvio (inclisiran), in collaboration with Novartis NVS to treat hypercholesterolemia in the EU. In the United States, it is approved to reduce low-density lipoprotein cholesterol. The drug’s label has also been expanded to cover high-risk cardiovascular patients and late-stage studies are underway to broaden its indication further. ALNY earns royalties from Novartis for Leqvio sales that add to the top line.

In the first half of 2025, Alnylam generated $236.8 million in net product revenues from its rare disease portfolio (Givlaari and Oxlumo), reflecting a 16% year-over-year increase. Expanding global adoption of these therapies is expected to sustain Alnylam’s top-line growth while diversifying its revenue streams and reducing reliance on Amvuttra.

ALNY’s Competition in the Market for Its Lead Drug

Alnylam’s push to broaden indications and expand the global reach of its marketed drugs is becoming increasingly critical as Amvuttra faces intensifying competition in the ATTR-CM market. Rival therapies, including Pfizer’s PFE Vyndaqel/Vyndamax (tafamidis) and BridgeBio’s BBIO Attruby (acoramidis), are already approved and competing for market share in this space.

Vyndaqel is one of the key in-line products that has driven improvement in Pfizer’s revenues in the first half of 2025. Global Vyndaqel family revenues of $3.1 billion rose 27% year over year in the first half of 2025, driven by continued demand growth due to increases in diagnosis and treatment rates, primarily in the United States and developed Europe. Pfizer’s Vyndaqel family includes global revenues from Vyndaqel as well as revenues for Vyndamax in the United States and Vynmac in Japan.

Approved in late 2024, Attruby is BridgeBio’s only marketed product. The drug generated sales worth $108.2 million in the first half of 2025, driven by solid uptake. BBIO reported that as of Aug. 1, 2025, 3,751 unique patient prescriptions for Attruby have been written by 1,074 unique healthcare providers since approval. BridgeBio is also currently evaluating acoramidis for the prevention of early-stage variant transthyretin amyloidosis in a late-stage study.

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