Onpattro Market Report 2026

Onpattro Market Overview

• The Onpattro market growth in the historic period has been driven by advancements in rna-based drug development, increased diagnosis of hATTR amyloidosis • Market expansion is supported by expansion of next-generation rna therapeutics, increasing investments in rare disease pipelines • Growth Driver: Impact Of Rising Prevalence Of Hereditary Transthyretin-Mediated Amyloidosis On The Onpattro Market Growth • Market Trend: Broadening Patient Access Through Strategic Drug Approvals • North America was the largest region in 2025 and Asia-Pacific is the fastest growing region.

What Is Covered Under Onpattro Market?

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 that cause organ damage. Onpattro is delivered via intravenous infusion and helps manage symptoms and improve the quality of life for affected patients. The main clinical indications of onpattro are 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 abnormal TTR protein production. These proteins accumulate as amyloid deposits in organs, most notably the heart and nerves, causing progressive damage. It is distributed through various distribution channels, such as direct sales, pharmacies, and online pharmacies, and used by several end users, including hospitals, specialty clinics, and home healthcare providers.

What Is The Onpattro Market Growth Forecast?

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.

Global Onpattro Market Segmentation

1) By Clinical Indication: Hereditary Transthyretin-Mediated Amyloidosis, Wild-Type Transthyretin Amyloidosis, Other Transthyretin-Related Amyloidoses 2) By Distribution Channel: Direct Sales, Pharmacies, Online Pharmacies 3) By End User: Hospitals, Specialty Clinics, Home Healthcare Providers

What Is The Driver Of The Onpattro Market?

The rising prevalence of hereditary transthyretin-mediated amyloidosis is expected to propel the growth of the onpattro market going forward. Hereditary transthyretin-mediated amyloidosis (hATTR) is a rare genetic disorder where mutated TTR proteins form harmful deposits, affecting nerves and the heart. The rising prevalence of hereditary transthyretin-mediated amyloidosis is driven by better diagnostics, genetic testing, and increasing awareness, identifying more cases. Onpattro is used to treat hereditary transthyretin-mediated amyloidosis by silencing the transthyretin (TTR) gene to reduce amyloid protein production and prevent organ damage. For instance, in February 2024, according to Verywell Health, a US-based health information platform, Globally, hereditary transthyretin-mediated amyloidosis affects 5,500–38,500 individuals, with U.S. estimates ranging from 104 to 3,847 cases of hereditary transthyretin-mediated amyloidosis with polyneuropathy, highlighting significant underdiagnosis. Therefore, the rising prevalence of hereditary transthyretin-mediated amyloidosis is driving the growth of the onpattro industry.

Key Players In The Global Onpattro Market

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

Global Onpattro Market Trends and Insights

The key trend in the onpattro market is focused on expanding the drug’s indications through supplemental drug applications, aiming to maximize market potential and meet a wider range of patient needs. Supplemental drug applications generally aim to extend the approved uses of a drug, such as targeting new indications, populations, or formulations, to broaden its therapeutic reach and market potential. 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 treatment targets cardiomyopathy associated with transthyretin amyloidosis (ATTR). ONPATTRO is an RNA interference (RNAi) therapeutic designed to specifically target the transthyretin (TTR) protein, which can accumulate abnormally in patients with ATTR amyloidosis, leading to severe complications such as cardiomyopathy. The drug was initially approved in 2018 for the treatment of polyneuropathy related to hereditary ATTR amyloidosis. The recent sNDA submission was based on results from the Phase III APOLLO-B study, which demonstrated that patisiran significantly improved functional capacity and quality of life in patients with cardiomyopathy compared to a placebo.

What Are Latest Mergers And Acquisitions In The Onpattro Market?

In March 2023, Alnylam Pharmaceuticals, a US-based biopharmaceutical company, partnered with Medison Pharma to expand the availability and commercialization of RNAi therapeutics in Central and Eastern Europe. This partnership aims to enhance the commercialization of Alnylam's RNAi therapeutics, including ONPATTRO (patisiran), which is designed for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) in adults with stage 1 or stage 2 polyneuropathy Medison Pharma is an Israel-based biopharmaceutical company that specializes in the commercialization and distribution of innovative therapies.

Regional Insights

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 this market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in this market report are Australia, India, China, South Korea, Japan, UK, France, Germany, USA, Canada.

What Defines the Onpattro Market?

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.

How is Market Value Defined and Measured?

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.

What Key Data and Analysis Are Included in the Onpattro Market Report 2026?

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