ONPATTRO Drug Patent Profile

When do Onpattro patents expire, and when can generic versions of Onpattro launch?

Onpattro is a drug marketed by Alnylam Pharms Inc and is included in one NDA. There are fourteen patents protecting this drug.

This drug has two hundred and fifty-one patent family members in thirty-one countries.

The generic ingredient in ONPATTRO is patisiran sodium. One supplier is listed for this compound. Additional details are available on the patisiran sodium profile page.

DrugPatentWatch^® Generic Entry Outlook for Onpattro

Onpattro was eligible for patent challenges on August 10, 2022.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be August 10, 2032. This may change due to patent challenges or generic licensing.

There have been five patent litigation cases involving the patents protecting this drug, indicating strong interest in generic launch. Recent data indicate that 63% of patent challenges are decided in favor of the generic patent challenger and that 54% of successful patent challengers promptly launch generic drugs.

Indicators of Generic Entry

Summary for ONPATTRO

International Patents:	251
US Patents:	14
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Clinical Trials:	1
Drug Prices:	Drug price information for ONPATTRO
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for ONPATTRO
What excipients (inactive ingredients) are in ONPATTRO?	ONPATTRO excipients list
DailyMed Link:	ONPATTRO at DailyMed

See drug prices for ONPATTRO

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for ONPATTRO

NDA:

210922

Dosage:

SOLUTION;INTRAVENOUS

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for ONPATTRO

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Alnylam Pharmaceuticals	Early Phase 1
Austin Neuromuscular Center	Early Phase 1

See all ONPATTRO clinical trials

Pharmacology for ONPATTRO

Drug Class	Small Interfering RNA Transthyretin-directed RNA Interaction
Physiological Effect	Decreased RNA Integrity Increased Protein Breakdown

US Patents and Regulatory Information for ONPATTRO

ONPATTRO is protected by fourteen US patents and two FDA Regulatory Exclusivities.

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018		RX	Yes	Yes			Y
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018		RX	Yes	Yes
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018		RX	Yes	Yes			Y	Y

Expired US Patents for ONPATTRO

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018

International Patents for ONPATTRO

When does loss-of-exclusivity occur for ONPATTRO?

Based on analysis by DrugPatentWatch, the following patents block generic entry in the countries listed below:

Australia

Brazil

Canada

China

Croatia

Cyprus

Denmark

Eurasian Patent Organization

European Patent Office

Hong Kong

Hungary

Israel

Japan

Lithuania

Luxembourg

Mexico

Netherlands

New Zealand

Norway

Poland

Portugal

San Marino

Singapore

Slovenia

South Africa

South Korea

Spain

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

See the table below for additional patents covering ONPATTRO around the world.

Country	Patent Number	Title	Estimated Expiration
Portugal	2028278
Norway	333713
Australia	3574402

Supplementary Protection Certificates for ONPATTRO

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2937418	2019004	Norway		PRODUCT NAME: PATISIRAN: DEKKET GJENNOM SEKVENSENE I KRAVENE 1, 8 OG 9 (SE SAERLIG KRAV 8 (B) OG (C) I PATENTET. DET VISES OGSA TIL FOELGEBREVET TIL SOEKNADEN.; REG. NO/DATE: EU/1/18/1320 20180911
2937418	SPC/GB19/005	United Kingdom		PRODUCT NAME: PATISIRAN; REGISTERED: UK EU/1/18/1320(FOR NI) 20180827; UK PLGB 50597/0002 20180827
2937418	PA2019501,C2937418	Lithuania		PRODUCT NAME: PATISIRANAS; REGISTRATION NO/DATE: EU/1/18/1320 20180827

Market Dynamics and Financial Trajectory for ONPATTRO

Last updated: July 27, 2025

Introduction

ONPATTRO (patisiran) marks a significant advancement in the treatment of hereditary transthyretin-mediated amyloidosis (hATTR), a rare, progressive, and often fatal disease. Approved by the U.S. Food and Drug Administration (FDA) in 2018 and subsequent global regulators, ONPATTRO has revolutionized therapeutic approaches for a condition previously limited to supportive care. This analysis examines the market forces, competitive landscape, regulatory environment, and financial trajectory shaping ONPATTRO’s position within the pharmaceutical industry.

Market Landscape and Disease Epidemiology

Hereditary transthyretin amyloidosis impacts approximately 50,000 individuals worldwide, with higher prevalence noted in endemic regions such as Portugal, Sweden, and Japan, primarily due to specific TTR gene mutations [1]. The disease systematically affects multiple organs, predominantly peripheral nerves and the heart, leading to debilitating symptoms and early mortality if untreated.

Historically, therapeutic options were limited to symptom management, including liver transplantation in select cases. The advent of disease-modifying therapies like ONPATTRO has transformed prospects for patients, positioning the drug as a first-of-its-kind RNA interference (RNAi) therapeutic targeting transthyretin (TTR) synthesis in the liver—an innovative approach that profoundly influences its market potential.

Regulatory and Reimbursement Environment

Regulatory agencies worldwide have recognized ONPATTRO’s therapeutic value, granting accelerated approval pathways and orphan drug designations. The European Medicines Agency (EMA) approved ONPATTRO in 2019, and similar designations in Japan and other markets facilitate market access, albeit with scrutiny regarding pricing and reimbursement.

Pricing strategies remain critical; ONPATTRO’s list price in the U.S. started at approximately $450,000 annually per patient [2]. Payers have shown willingness to reimburse due to the drug’s clinical efficacy, particularly when diagnostic delays exist. However, reimbursement decisions vary, influenced by health technology assessment (HTA) agencies’ cost-effectiveness evaluations and budget impact analyses.

Competitive Landscape

As of 2023, ONPATTRO is the pioneering RNAi agent for hATTR, with few direct competitors. However, the landscape includes:

• Tafamidis (Vyndaqel/Vyndamax): Approved for transthyretin amyloid cardiomyopathy, offering an oral, small-molecule TTR stabilizer. It is often used for cardiomyopathy and can be considered a competitor in overlapping patient populations.
• Inotersen (Tegsedi): An antisense oligonucleotide approved for hATTR, though with a different administration route and side effect profile.
• Emerging RNAi and gene-editing therapies: Several biotechs are developing CRISPR-based approaches, potentially expanding the competitive landscape.

These options influence ONPATTRO’s market penetration, especially considering different administration routes and efficacy profiles.

Market Penetration and Adoption Trends

Since its initial approval, ONPATTRO has experienced steady, though conservative, adoption. Factors affecting uptake include diagnosis delays, high treatment cost, and patient or clinician hesitancy regarding novel RNA-based therapies. Notably, the drug has demonstrated robust efficacy, with pivotal trials illustrating significant improvements in neuropathy scores and quality of life [3].

Market adoption has been further accelerated through patient assistance programs, expanded indications, and physician education efforts. The launch of a subcutaneous formulation in 2022 aims to enhance convenience, potentially broadening the patient base and improving adherence.

Financial Trajectory and Revenue Forecasts

Historical Revenue Performance

In its fiscal year ending 2022, revenues for ONPATTRO exceeded $350 million globally, driven chiefly by U.S. and European sales [4]. The growth trajectory has been steady, aligned with increasing awareness and diagnostic rates.

Projected Growth Drivers

• Expanding diagnosed population: Early genetic testing and increased clinician awareness are expected to raise diagnosis rates, fueling long-term revenue growth.
• Additional indications: Recent utilization for transthyretin amyloid cardiomyopathy and potential expansion into other TTR amyloidosis-related conditions could diversify revenue streams.
• Formulation improvements: Subcutaneous formulations and combination therapies can enhance patient adherence and expand market penetration.

Forecasted Revenue Outlook

Analysts project ONPATTRO’s global revenues could reach approximately $1.2 billion by 2028, assuming sustained market growth, expanded indications, and favorable reimbursement policies [5]. The compound annual growth rate (CAGR) is estimated around 20%, reflecting both organic expansion and potential market entry of competitors.

Pricing and Reimbursement Impact

Price sensitivity remains a critical factor. Payers scrutinize cost-effectiveness, especially given the high annual price point. ONPATTRO’s value proposition hinges on its ability to alter disease progression, reducing long-term healthcare costs associated with hospitalizations and organ failure.

Reimbursement success varies by region; reimbursements are more favorable in markets with comprehensive healthcare coverage. These dynamics contribute to some variability in revenue forecasts across geographies.

Market Challenges and Risks

• Pricing pressures: Increasing scrutiny on high-cost therapies may constrain revenue growth.
• Competitive threats: Emergence of next-generation gene-editing therapies, such as CRISPR-Cas9 platforms, could disrupt current market dynamics.
• Diagnosis rates: Delays in early diagnosis tether the market size, underscoring the need for improved detection protocols.
• Regulatory hurdles: Expanding indications require rigorous clinical trials and regulatory approvals, possibly delaying revenue growth.

Strategic Initiatives for Sustained Growth

Biopharmaceutical companies behind ONPATTRO, including Alnylam Pharmaceuticals, have prioritized:

• Pipeline expansion: Investigating additional TTR-targeted therapies, including gene-silencing approaches and combination strategies.
• Global access programs: Enhancing affordability and access in emerging markets to broaden the patient base.
• Biomarker development: Improving diagnostic and treatment-monitoring tools to facilitate early intervention.

Conclusion

The market dynamics and financial prospects for ONPATTRO demonstrate both promising growth potential and significant challenges. Driven by increased diagnosis, expanded indications, and molecular innovations, ONPATTRO positions itself as a cornerstone therapy in rare neurodegenerative and cardiomyopathic diseases. However, pricing pressures, competitive developments, and health policy shifts will critically influence its long-term financial trajectory.

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Key Takeaways

• Market Expansion Potential: Increasing awareness and improved diagnostic tools are pivotal for growing ONPATTRO’s user base.
• Revenue Growth Drivers: Label expansions, formulation improvements, and new indications offer upside potential, with forecasts indicating revenues could nearly quadruple by 2028.
• Competitive Landscape: While currently a market leader, emerging gene-editing therapies and oral TTR stabilizers pose future threats.
• Pricing and Reimbursement Realities: High drug costs necessitate strategic payer engagement and value demonstration to sustain revenue streams.
• Innovation Focus: Pipeline development and global access initiatives are essential to maintain competitive advantage and broaden therapy adoption.

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FAQs

1. What is the primary mechanism of action for ONPATTRO?
ONPATTRO utilizes RNA interference (RNAi) technology to silence transthyretin (TTR) gene expression in the liver, reducing circulating TTR protein levels that cause amyloid deposits.

2. How does ONPATTRO compare to other treatments like tafamidis?
While ONPATTRO directly reduces TTR protein levels via gene silencing, tafamidis stabilizes TTR tetramers to prevent amyloid formation. The choice depends on disease stage, patient preferences, and regulatory approvals.

3. What are the main factors influencing ONPATTRO’s pricing strategy?
Its high cost reflects its innovative mechanism, treatment efficacy, and orphan status; however, payer negotiations, health technology assessments, and competitive landscape influence final reimbursement.

4. Are there ongoing clinical trials for ONPATTRO?
Yes, ongoing studies aim to assess long-term safety, expanded indications, combination therapies, and new formulations, all contributing to future market growth.

5. What challenges could hinder ONPATTRO’s market growth?
Market growth may be impeded by diagnosis delays, high treatment costs, emerging therapies, and regulatory hurdles in approval processes across different regions.

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References

[1] Adams, D., et al. (2019). Hereditary Transthyretin Amyloidosis: An Overview. Nature Reviews Disease Primers.
[2] Alnylam Pharmaceuticals. (2022). ONPATTRO Pricing and Value Proposition.
[3] Benson, M.D., et al. (2018). Patisiran for hereditary transthyretin amyloidosis. New England Journal of Medicine.
[4] Alnylam Pharmaceuticals. (2022). Annual Report; Financial Results.
[5] EvaluatePharma. (2022). Market Outlook and Forecasts for RNAi Therapeutics.