Onpattro Market Report 2025: Epidemiology, Pipeline Analysis, Market Insights & Forecasts

This Onpattro market report delivers an in-depth analysis of the market’s key characteristics, including size, growth potential, and segmentation. It provides a detailed breakdown of the market across major regions and leading countries, highlighting historical data and future growth projections. The report also examines the competitive landscape, market share insights, emerging trends, and strategic developments shaping the market.

The growth observed during the historic period can be attributed to the increasing prevalence of hereditary transthyretin-mediated amyloidosis, heightened awareness of rare diseases, greater investments in research and development, an aging population, and rising healthcare spending.

The anticipated growth during the forecast period is driven by factors such as collaborations and licensing agreements, regulatory support, a rising incidence of chronic diseases, expanding biopharmaceutical partnerships, and a growing number of transthyretin amyloidosis cases. Key trends expected to shape the market during this period include the increasing use of ribonucleic acid-based therapies, a rise in orphan drug designations, the integration of digital health tools, a shift toward personalized medicine, and advancements in drug delivery technologies.

The increasing prevalence of hereditary transthyretin-mediated amyloidosis is expected to drive the growth of the onpattro market in the future. Hereditary transthyretin-mediated amyloidosis (hATTR) is a rare genetic disorder where mutated TTR proteins form harmful deposits, affecting the nerves and heart. The rising prevalence of hATTR is fueled by advancements in diagnostics, genetic testing, and increased awareness, which are helping identify more cases. Onpattro treats hereditary transthyretin-mediated amyloidosis by silencing the transthyretin (TTR) gene to reduce amyloid protein production and prevent organ damage. For example, in February 2024, Verywell Health, a U.S.-based health information platform, reported that globally, hereditary transthyretin-mediated amyloidosis affects 5,500 to 38,500 people, with estimates in the U.S. ranging from 104 to 3,847 cases of hereditary transthyretin-mediated amyloidosis with polyneuropathy, highlighting significant underdiagnosis. As a result, the growing prevalence of hereditary transthyretin-mediated amyloidosis is fueling the expansion of the onpattro market.

A key trend in the onpattro market is the expansion of the drug’s indications through supplemental drug applications, aiming to maximize its market potential and address a broader range of patient needs. Supplemental drug applications are typically intended to extend the approved uses of a drug, such as targeting new indications, patient populations, or formulations, to enhance its therapeutic scope and market reach. For instance, in February 2023, Alnylam Pharmaceuticals, a U.S.-based pharmaceutical company, announced the acceptance of a Supplemental New Drug Application (sNDA) for ONPATTRO (patisiran). This treatment targets cardiomyopathy associated with transthyretin amyloidosis (ATTR). ONPATTRO is an RNA interference (RNAi) therapeutic that specifically targets the transthyretin (TTR) protein, which can accumulate abnormally in patients with ATTR amyloidosis, leading to serious complications such as cardiomyopathy. The drug was initially approved in 2018 for treating polyneuropathy related to hereditary ATTR amyloidosis. The recent sNDA submission was based on results from the Phase III APOLLO-B study, 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, a U.S.-based biopharmaceutical company, formed a partnership with Medison Pharma to expand the availability and commercialization of RNAi therapeutics in Central and Eastern Europe. This collaboration aims to enhance the commercialization of Alnylam’s RNAi therapeutics, including ONPATTRO (patisiran), which is used to treat hereditary transthyretin-mediated amyloidosis (hATTR) in adults with stage 1 or stage 2 polyneuropathy. Medison Pharma, an Israel-based biopharmaceutical company, specializes in the commercialization and distribution of innovative therapies.

The key company operating in the onpattro market is Alnylam Pharmaceuticals Inc.

North America was the largest region in the onpattro market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in onpattro report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa. The countries covered in the onpattro market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Onpattro (patisiran) is an FDA-approved treatment for hereditary transthyretin-mediated amyloidosis (hATTR), a rare genetic condition. The medication works by targeting and silencing the transthyretin (TTR) gene, reducing the production of amyloid proteins that cause organ damage. Administered via intravenous infusion, Onpattro helps alleviate symptoms and improve the quality of life for patients with this condition.

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 TTR gene, resulting in abnormal TTR protein production. These proteins form amyloid deposits in organs, primarily affecting the heart and nerves, leading to progressive damage. Onpattro is distributed through various channels, including direct sales, pharmacies, and online pharmacies, and is used by various healthcare providers, such as hospitals, specialty clinics, and home healthcare services.

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.

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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