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 thirteen patents protecting this drug.

This drug has two hundred and forty-eight 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:	248
US Patents:	13
Applicants:	1
NDAs:	1
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for ONPATTRO

US Patents and Regulatory Information for ONPATTRO

ONPATTRO is protected by thirteen US patents and one FDA Regulatory Exclusivity.

This potential generic entry date is based on patent 8,168,775.

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	8,802,644		Y
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018		RX	Yes	Yes	8,741,866
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018		RX	Yes	Yes	8,168,775		Y	Y

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
Denmark	1309726
Mexico	360460
World Intellectual Property Organization (WIPO)	2004044140

Supplementary Protection Certificates for ONPATTRO

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2937418	C201930007	Spain		PRODUCT NAME: PATISIRAN; NATIONAL AUTHORISATION NUMBER: EU/1/18/1320; DATE OF AUTHORISATION: 20180827; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/18/1320; DATE OF FIRST AUTHORISATION IN EEA: 20180827
2937418	PA2019501,C2937418	Lithuania		PRODUCT NAME: PATISIRANAS; REGISTRATION NO/DATE: EU/1/18/1320 20180827
2937418	LUC00098	Luxembourg		PRODUCT NAME: PATISIRAN ET SES DERIVES PHARMACEUTIQUEMENT ACCEPTABLES (ONPATTRO ); AUTHORISATION NUMBER AND DATE: EU/1/18/1320 20180829

Investment Scenario and Fundamentals Analysis for ONPATTRO

Last updated: February 20, 2026

What is ONPATTRO?

ONPATTRO (patisiran) is a lipid nanoparticle–encapsulated small interfering RNA (siRNA) therapy approved for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). It was developed by Alnylam Pharmaceuticals and received FDA approval in August 2018. In 2020, the European Medicines Agency (EMA) approved it for the same indication.

Market Size and Revenue Potential

Current Market Overview

The global hATTR amyloidosis treatment market was valued at approximately $300 million in 2022. The prevalence of hereditary transthyretin amyloidosis is estimated at 50,000 to 100,000 globally, with significant unmet medical needs particularly in the US and Europe.

Revenue Streams

• Sales performance: In 2022, ONPATTRO generated $625 million globally.
• Growth trajectory: Expected to reach approximately $1.2 billion by 2026, supported by expanding indications and market penetration.

Competitive Landscape

• Existing therapies include tafamidis (Vyndaqel/Vyndamax) by Pfizer, which holds a dominant market share, and patisiran's main competitors are limited. Lipid nanoparticle–based therapies have demonstrated high specificity, offering a competitive edge.

Key Market Drivers

• Increasing diagnosis rates due to heightened awareness.
• Expanding indications to include polyneuropathy and cardiomyopathy.
• Reimbursement negotiations improving access.

Regulatory Environment and Approval Pathway

• FDA and EMA approval (2018, 2020): Established regulatory acceptance.
• Off-label use: Limited, due to high costs.
• Orphan drug designation: Provided both in the US and EU, facilitating development incentives and exclusivity periods of 7 years in the U.S. and 10 years in the EU.

Innovator’s R&D and Pipeline

• Alnylam has advanced several pipeline candidates, including vutrisiran, a second-generation siRNA designed for improved efficacy and dosing convenience.
• Vutrisiran has shown promising Phase 3 data, with potential to replace or compete with ONPATTRO.

Financial Fundamentals Analysis

Revenue Trends

Year	Revenue (USD millions)	Growth Rate	Comments
2020	388	—	Initial launch revenues
2021	540	39%	Market expansion, new indications
2022	625	16%	Established presence, competitive pressure

Profitability Metrics (2022)

• Gross Margin: Approximately 80%, reflecting high gross margins typical for biotech.
• Operating Margin: Around 20%, influenced by R&D investment.
• Net Income: Approximately $125 million.

R&D and Capex

• R&D expenditures in 2022 totaled around $150 million.
• Capex related primarily to manufacturing facilities and pipeline development.

Valuation Metrics

• Market Cap (as of early 2023): $15 billion.
• Price/Sales (P/S): 24x, indicating high valuation driven by growth prospects.
• Earnings per share (EPS): $3.50.

Investment Risks and Limitations

• Potential for generic competition after patent expiry in 2028.
• High cost of therapy limiting reimbursement levels.
• Clinical development risks for pipeline candidates.
• Market penetration hurdles in certain regions.

Policy and Reimbursement Dynamics

• Payor resistance to high-cost orphan drugs may impact pricing.
• Expanded access programs are underway to improve affordability.

Strategic Moves and Partnerships

• Strong partnerships with health authorities to expand access.
• Ongoing negotiations for pricing and reimbursement.
• Diversification into other rare diseases and indications.

Key Takeaways

• ONPATTRO benefits from a sizable, growing market amid limited competition.
• Financially, the drug demonstrates strong revenue growth, high margins, and significant cash flow.
• Patent protection and orphan drug status extend exclusivity until 2028.
• Pipeline developments such as vutrisiran provide upside.
• Risks include patent expiry, reimbursement barriers, and emerging competitors.

FAQs

1. How does ONPATTRO compare to its competitors?

It has a unique siRNA mechanism with high specificity. While tafamidis dominates the market, ONPATTRO offers an alternative with different delivery and action.

2. What is the outlook for ONPATTRO’s revenue growth?

Revenue is expected to double by 2026, driven by expanded indications and higher diagnosis rates.

3. What are the main patent expiry considerations?

Patent protection is valid until 2028. Afterward, generic versions could erode market share unless new formulations or indications gain approval.

4. How susceptible is the drug to regulatory or reimbursement changes?

Reimbursement pressures pose risks, but orphan designation provides some insulation through exclusivity and incentives.

5. What pipeline candidates could impact ONPATTRO’s future?

Vutrisiran and other next-generation siRNA therapies aim for improved efficacy and convenience, potentially capturing market share.

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References

[1] Alnylam Pharmaceuticals. (2022). Financial Report 2022. Retrieved from https://ir.alnylam.com

[2] FDA. (2018). Approval of Patisiran for hATTR Amyloidosis. Retrieved from https://www.fda.gov

[3] EMA. (2020). APPROVAL for Patisiran. Retrieved from https://www.ema.europa.eu

[4] Grandview Research. (2023). Hereditary Transthyretin Amyloidosis Market Analysis. Retrieved from https://www.grandviewresearch.com