Onpattro Market Report 2026: Epidemiology, Pipeline Analysis, Market Insights & Forecasts

The growth in the historic period can be attributed to advancements in rna-based drug development, increased diagnosis of hATTR amyloidosis, regulatory approvals for gene-silencing therapies, expansion of specialty infusion centers, clinical validation of patisiran efficacy.

The growth in the forecast period can be attributed to expansion of next-generation rna therapeutics, increasing investments in rare disease pipelines, growing focus on personalized medicine, development of subcutaneous delivery alternatives, rising adoption of genetic screening programs. Major trends in the forecast period include increasing adoption of rna interference therapies, rising focus on rare genetic disease treatment, expansion of precision gene silencing approaches, improved clinical outcomes in amyloidosis care, growing integration of advanced infusion therapies.

The increasing prevalence of hereditary transthyretin-mediated amyloidosis (hATTR) is expected to drive the growth of the Onpattro market in the coming years. hATTR is a rare genetic disorder in which mutated TTR proteins form harmful deposits that affect the heart and nerves. The rise in the prevalence of this condition is attributed to advancements in diagnostics, genetic testing, and growing awareness, which have led to more cases being identified. Onpattro is used to treat hATTR by silencing the TTR gene to reduce amyloid protein production and prevent further organ damage. For example, in February 2024, according to Verywell Health, a US-based health information platform, hereditary transthyretin-mediated amyloidosis affects between 5,500 and 38,500 individuals globally, with U.S. estimates ranging from 104 to 3,847 cases of hATTR with polyneuropathy, indicating significant underdiagnosis. Therefore, the rising prevalence of hATTR is fueling the growth of the Onpattro market.

A key trend in the Onpattro market is the expansion of its indications through supplemental drug applications, aimed at maximizing its market potential and addressing a broader range of patient needs. Supplemental drug applications are designed to extend the approved uses of a drug, such as targeting new indications, populations, or formulations, to expand its therapeutic reach. For instance, in February 2023, Alnylam Pharmaceuticals, a US-based pharmaceutical company, announced the acceptance of a Supplemental New Drug Application (sNDA) for Onpattro (patisiran). This application aims to target cardiomyopathy associated with transthyretin amyloidosis (ATTR). Initially approved in 2018 for the treatment of polyneuropathy related to hereditary ATTR amyloidosis, Onpattro's recent sNDA submission was based on Phase III APOLLO-B study results, which showed that patisiran significantly improved functional capacity and quality of life in patients with cardiomyopathy compared to a placebo.

In March 2023, Alnylam Pharmaceuticals partnered with Medison Pharma, an Israel-based biopharmaceutical company, to expand the availability and commercialization of RNA interference (RNAi) therapeutics in Central and Eastern Europe. This partnership aims to enhance the commercialization of Onpattro and other RNAi therapeutics for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) in adults with stage 1 or stage 2 polyneuropathy.

Major companies operating in the onpattro market are Alnylam Pharmaceuticals Inc.

North America was the largest region in the onpattro market in 2025. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the onpattro market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the onpattro market report are Australia, India, China, South Korea, Japan, UK, France, Germany, USA, Canada.

Tariffs are impacting the onpattro market by increasing costs of imported lipid nanoparticles, nucleic acid raw materials, sterile manufacturing equipment, and cold-chain infusion logistics. North America and Europe are most affected due to reliance on advanced global RNA supply chains, while emerging markets face affordability barriers. These tariffs elevate therapy costs and slow market penetration. However, they are also encouraging localized RNA manufacturing, domestic formulation capabilities, and long-term supply chain strengthening for advanced gene-silencing medicines.

The onpattro market research report is one of a series of new reports that provides onpattro market statistics, including onpattro industry global market size, regional shares, competitors with a onpattro market share, detailed onpattro market segments, market trends and opportunities, and any further data you may need to thrive in the onpattro industry. This onpattro market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.

Onpattro (patisiran) is an FDA-approved medication used to treat hereditary transthyretin-mediated amyloidosis (hATTR), a rare genetic disorder. It works by targeting and silencing the transthyretin (TTR) gene to reduce the production of amyloid proteins, which cause organ damage. Onpattro is administered via intravenous infusion and helps manage symptoms, improving the quality of life for affected patients.

The primary clinical indications for Onpattro include hereditary transthyretin-mediated amyloidosis, wild-type transthyretin amyloidosis, and other transthyretin-related amyloidoses. Hereditary transthyretin-mediated amyloidosis is a genetic disorder caused by mutations in the transthyretin (TTR) gene, leading to the production of abnormal TTR proteins. These proteins accumulate as amyloid deposits in organs, particularly in the heart and nerves, causing progressive damage. Onpattro is distributed through various channels, such as direct sales, pharmacies, and online pharmacies, and is used by a range of end users, including hospitals, specialty clinics, and home healthcare providers.

The onpattro market consists of sales of lipids, drug delivery systems, and supportive diagnostic tools. Values in this market are ‘factory gate’ values, that is, the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

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