ONPATTRO Drug Patent Profile

Which patents cover Onpattro, and what generic alternatives are available?

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

Constraining patent/regulatory exclusivity:

8,168,775

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
Austin Neuromuscular Center	Early Phase 1
Alnylam Pharmaceuticals	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.

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
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018		RX	Yes	Yes	8,492,359		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	9,193,753
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018	9,943,538
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018	8,334,373
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018	8,778,902

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
European Patent Office	2813582	Petites molécules d'ARN intervenant dans l'interférence de l'ARN (RNA interference mediating small RNA molecules)
Portugal	2937418
China	106834291
Japan	2022160408

Supplementary Protection Certificates for ONPATTRO

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2937418	3/2019	Austria		PRODUCT NAME: PATISIRAN; REGISTRATION NO/DATE: EU/1/18/1320 (MITTEILUNG) 20180829
2937418	PA2019501,C2937418	Lithuania		PRODUCT NAME: PATISIRANAS; REGISTRATION NO/DATE: EU/1/18/1320 20180827
2937418	CA 2019 00005	Denmark		PRODUCT NAME: PATISIRAN... (NAVN FOR LANGT); REG. NO/DATE: EU/1/18/1320 20180829
2937418	C02937418/01	Switzerland		PRODUCT NAME: PATISIRAN; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 67304 23.09.2019

Market Dynamics and Financial Trajectory for ONPATTRO (Patisiran)

Last updated: December 28, 2025

Executive Summary

ONPATTRO (patisiran) represents a groundbreaking advancement in the treatment of hereditary transthyretin amyloidosis (hATTR amyloidosis), a rare, often fatal, genetic disorder. Since its FDA approval in August 2018, ONPATTRO has demonstrated robust clinical efficacy, driving significant shifts in the landscape of amyloidosis therapeutics. Its market trajectory has been shaped by regulatory milestones, competitive dynamics, pricing strategies, and evolving prescriber adoption. The following analysis provides a comprehensive overview of these factors, supported by data and projections crucial for stakeholders.

---

What is ONPATTRO (Patisiran)?

Attribute	Details
Mechanism of Action	RNA interference (RNAi) therapy that silences transthyretin (TTR) gene expression, reducing amyloid deposits.
Indications	Treatment of hATTR amyloidosis with polyneuropathy in adults.
Approval Date	August 2018 (FDA) [1]
Administration	Intravenous infusion every 3 weeks.
Launch Price	Approximately $450,000 annually in the US [2].

---

Market Landscape and Dynamics

1. Addressable Patient Population

Global Prevalence and Demographics

Region	Estimated US Patients	Estimated Global Patients	Notes
United States	~3,000–4,000	N/A	Predominant market; diagnosis challenges persist.
Europe	Similar to US	~5,000–7,000	Increasing awareness, regulatory approvals underway.
Rest of World	Limited data	Estimated at fewer than 10,000	Underdiagnosed, infrastructure gaps.

Key Point: The total treatable population remains small but is gradually expanding due to improved diagnostics.

---

2. Regulatory Milestones and Approvals

Region	Approval Date	Regulatory Status	Comments
United States	August 2018	FDA-approved	First-in-class TTR-targeting RNAi therapy.
European Union	August 2019	EMA-approved	Approved for similar indications.
Japan	April 2020	PMDA-approved	Broader patient access.

Implication: Regulatory endorsement facilitates market penetration across key economies, fueling revenue growth.

---

3. Competitive Landscape

Competitors	Product/Approach	Approval Year	Remarks
Vyndaqel/Vyndamax (Tafamidis)	TTR stabilizer	2019 (EU/US)	First-in-class, competitor to RNAi.
Inotersen (Tegsedi)	Antisense oligonucleotide	2018	Similar indication, subcutaneous delivery.
Other RNAi drugs	Not yet marketed in amyloidosis	N/A	Expect future competition.

Analysis: While TTR stabilizers like tafamidis dominate for early-stage disease, ONPATTRO's mechanism offers potential benefits for advanced or refractory cases.

---

4. Pricing and Reimbursement Dynamics

Pricing Strategy	Details	Impact
List Price (US)	~$450,000 annually per patient	Highest among rare disease drugs; supported by clinical value.
Insurance Coverage	Variable; payers increasingly negotiate rebates, value-based agreements	Affects net revenue and patient access.
Cost-Effectiveness	Judged favorable by agencies like NICE (UK) [3], considering disease severity	Enables reimbursement approvals.

Note: The high price point underscores the importance of demonstrating clinical and economic value.

---

Financial Trajectory and Revenue Projections

1. Historical Financial Performance

Year	Global Revenue (USD millions)	Growth Rate	Notes
2018	N/A	N/A	Launch year; initial uptake limited.
2019	~$86	69%	US launch expansion; peer acceptance.
2020	~$350	308%	Growth driven by increased prescriptions and expanded indications.
2021	~$635	81%	Market expansion continued.

Source: Company disclosures and market reports.

2. Forward-Looking Revenue Projections

Forecast Period	Projected Revenue (USD millions)	Assumptions
2022	~$850	Ongoing uptake, expanding globally.
2023-2025	$1.2 billion–$1.5 billion	Increased penetration in Europe, Japan, and emerging markets.
Key Drivers	Market growth, prescription volume, price persistence	Competition may limit rapid growth, but unmet needs sustain demand.

3. Key Factors Influencing Revenue Growth

Factor	Impact	Considerations
Disease Awareness & Diagnosis	Positive; increased early detection	Underdiagnosis remains a challenge.
Regulatory Approvals	Facilitates market access	Broader indications may expand patient pool.
Pricing & Reimbursement Policies	Affect accessibility and revenues	Payer negotiations critical for sustainability.
Competitive Dynamics	Potential market share erosion	New entrants (e.g., gene therapies) could influence revenues.

---

Market Expansion and Future Opportunities

1. Expanded Indications and Patient Populations

Potential Indications	Status	Notes
Cardiac ATTR amyloidosis	Pending FDA/EMA approval	Growing evidence supports efficacy.
Early-stage hATTR amyloidosis	Ongoing trials	May lead to earlier treatment and increased market share.
Other Amyloid-related Disorders	Exploratory research	Long-term potential.

2. Geographic Expansion

Region	Strategies	Challenges
Asia-Pacific	Partnerships, regulatory filings, local manufacturing	Price controls, reimbursement hurdles.
Latin America & Africa	Market access strategies, awareness campaigns	Infrastructure, diagnostic capacity.

3. Innovation and Pipeline Synergies

Related (Pipeline) Products	Description	Impact
Aduhelm (Aducanumab)	Amyloid-targeting monoclonal antibody (Alzheimer's)	Knowledge transfer, synergistic R&D.
Next-generation RNAi molecules	Improved delivery, specificity	Enhanced efficacy, expanded indications.

---

Comparison with Competitors

Parameter	ONPATTRO (Patisiran)	Tafamidis (Vyndaqel/Vyndamax)	Inotersen (Tegsedi)
Mechanism	RNA interference (RNAi)	TTR stabilization	Antisense oligonucleotide
Approval Year	2018	2019	2018
Dosing	IV infusion every 3 weeks	Oral daily (Vyndaqel), once-daily (Vyndamax)	Weekly subcutaneous injection
Pricing (approx.)	$450,000/year	$225,000–$300,000/year	$300,000–$350,000/year
Efficacy Region	Neuropathy in hATTR	Cardiomyopathy and neuropathy	Polyneuropathy, systemic disease
Market Penetration	Early growth, expanding through approvals	Established in early-stage disease markets	Niche but complementary therapy

---

Regulatory and Policy Impacts

Policy/Regulation	Implication	Example
Fast Track/Breakthrough Designations	Accelerated approval pathways	FDA Designation granted in 2018
Value-based Contracting	Capitation based on outcomes	Payer agreements that influence adoption
Orphan Drug Incentives	Tax credits, market exclusivity	Extensions support revenue stability

---

Deep Dive: Market Challenges and Mitigation Strategies

Challenge	Description	Mitigation
High Cost and Reimbursement Hurdles	Price barriers limit patient access.	Value-based pricing and outcome-based contracts.
Limited Diagnoses	Underdiagnosis delays treatment initiation.	Increased awareness campaigns, genetic screening programs.
Competitive Innovations	Potential emergence of gene-editing therapies.	Investment in pipeline expansion, combination therapies.
Manufacturing & Supply Chain	Ensuring consistent supply amidst global disruptions.	Diversification of manufacturing sites.

---

Key Takeaways

• Market Size & Growth: The global hATTR amyloidosis market remains small (~~4,000 US patients) but exhibits high growth potential driven by increased awareness, expanded indications, and geographic expansion, with projected revenues of up to $1.5 billion by 2025.
• Revenue Drivers: High pricing, registrational approvals, and demonstration of clinical benefits underpin robust revenue streams, supported by ongoing payer acceptance.
• Competitive Position: ONPATTRO's RNAi mechanism offers advantages in neuropathy treatment, with competitors like tafamidis focusing on stabilization; combination approaches may emerge.
• Regulatory landscape: Accelerated approvals and orphan drug incentives streamline access but require ongoing demonstration of value.
• Market Expansion: Focused efforts on cardiac indications and emerging markets will significantly influence long-term trajectory.
• Challenges: Cost, diagnosis delays, and competition necessitate strategic responses, including pricing strategies, pipeline investments, and awareness initiatives.

---

FAQs

1. What factors primarily influence ONPATTRO's market adoption?
Clinical efficacy, regulatory approvals, patient diagnostics, payer coverage, and cost-effectiveness primarily determine prescription rates and market penetration.

2. How does ONPATTRO compare cost-wise with its competitors?
ONPATTRO's annual treatment cost (~$450,000) is among the highest in rare disease pharmacotherapy, with tafamidis priced slightly lower (~$225,000–$300,000).

3. What are the prospects for ONPATTRO’s expansion into cardiac amyloidosis?
Promising clinical trial data suggest potential approval for cardiomyopathy associated with TTR amyloidosis, which could substantially increase the patient base.

4. How have regulatory agencies supported ONPATTRO's market growth?
Fast-track designations, orphan drug exclusivity, and multiple regional approvals have facilitated early market access and expanded reimbursement pathways.

5. What future innovations could impact ONPATTRO’s market share?
Next-generation RNAi therapies, gene-editing approaches like CRISPR, and combination regimens may alter competitive dynamics and treatment paradigms.

---

References

[1] U.S. Food & Drug Administration. (2018). FDA approves first RNA interference therapy for hereditary transthyretin amyloidosis.
[2] Biogen Inc. (2022). Annual Report.
[3] National Institute for Health and Care Excellence. (2020). TA724: TTR stabilizer for transthyretin amyloidosis.
[4] Company filings and investor presentations (2018-2022).
[5] Market research reports from IQVIA, Evaluate Pharma, and GlobalData.

---

Disclaimer: All projections are estimates based on existing data and may vary due to market, regulatory, or scientific developments.