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Onpattro Market Size, Value And Growth Trajectory Through 2030
The growth observed in the historical period was attributable to advancements in RNA-based drug development, a heightened diagnosis rate of hATTR amyloidosis, regulatory authorizations for gene-silencing therapies, an expansion of specialty infusion centers, and the clinical validation of patisiran’s efficacy.
The anticipated expansion during the forecast period is primarily fueled by the proliferation of next-generation RNA therapeutics, increasing financial commitments to rare disease pipelines, a heightened focus on personalized medicine, the development of alternative subcutaneous delivery methods, and the rising uptake of genetic screening programs. Concurrently, key trends identified for this period encompass the wider adoption of RNA interference therapies, a greater emphasis on treating rare genetic disorders, the broadening implementation of precision gene silencing approaches, enhanced clinical outcomes in amyloidosis care, and the expanding integration of advanced infusion therapies.
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Onpattro Market Development Factors: What’s Supporting Demand?
The increasing occurrence of hereditary transthyretin-mediated amyloidosis is projected to drive the expansion of the onpattro market in the future. This condition, known as hATTR, is a rare genetic illness where mutated TTR proteins form harmful deposits, impacting both nerves and the heart. The growing prevalence of hereditary transthyretin-mediated amyloidosis is attributed to improved diagnostic methods, genetic screening, and heightened public awareness, leading to the identification of more cases. Onpattro serves as a treatment for hereditary transthyretin-mediated amyloidosis by silencing the transthyretin (TTR) gene, thereby reducing amyloid protein production and preventing organ damage. For example, in February 2024, according to Verywell Health, a US-based health information platform, hereditary transthyretin-mediated amyloidosis affects 5,500–38,500 individuals globally. U.S. estimates for hereditary transthyretin-mediated amyloidosis with polyneuropathy range from 104 to 3,847 cases, indicating significant underdiagnosis. Consequently, the rising prevalence of hereditary transthyretin-mediated amyloidosis is fueling the growth of the onpattro market.
Onpattro Market Segment Analysis And Revenue Potential
The onpattro market covered in this report is segmented –
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
Onpattro Market Transformation Trends: What Innovations Are Driving Change?
The central trend in the onpattro market focuses on expanding the drug’s approved applications via supplemental drug applications, intending to maximize market potential and cater to a wider array of patient needs. Supplemental drug applications generally aim to extend the authorized uses of a medication, such as addressing new indications, patient populations, or formulations, to broaden its therapeutic scope and market reach. For example, in February 2023, Alnylam Pharmaceuticals, a US-based pharmaceutical company, announced the acceptance of a Supplemental New Drug Application (sNDA) for ONPATTRO (patisiran). This therapy targets cardiomyopathy associated with transthyretin amyloidosis (ATTR). ONPATTRO functions as 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 initially received approval in 2018 for the treatment of polyneuropathy related to hereditary ATTR amyloidosis. The recent sNDA submission was based on findings from the Phase III APOLLO-B study, which indicated that patisiran notably improved functional capacity and quality of life in cardiomyopathy patients compared to a placebo.
Onpattro Market Key Players Shaping Industry Direction
Major companies operating in the onpattro market are Alnylam Pharmaceuticals Inc.
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Onpattro Market Top Region By Revenue And Market Share
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.
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