ONPATTRO Drug Patent Profile

Which patents cover Onpattro, 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 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 four 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:	250
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
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018		RX	Yes	Yes	11,079,379		Y	Y
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018		RX	Yes	Yes

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	8,552,171
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018	8,778,902
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018	8,895,718
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018	8,362,231
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018	9,943,539
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018	8,895,721

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
Australia	2023248138
European Patent Office	3354733	COMPOSITIONS ET PROCÉDÉS PERMETTANT D'INHIBER L'EXPRESSION DU GÈNE EG5 (COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TRANSTHYRETIN)
Israel	310600
Japan	2015119713	ＲＮＡ干渉のＲＮＡ配列特異的メディエータ (RNA SEQUENCE-SPECIFIC MEDIATORS OF RNA INTERFERENCE)
Singapore	10201809460S	COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TRANSTHYRETIN
South Korea	20080023768

Supplementary Protection Certificates for ONPATTRO

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2937418	122018000133	Germany		PRODUCT NAME: PATISIRAN; REGISTRATION NO/DATE: EU/1/18/1320 20180827
1407044	132019000000031	Italy		PRODUCT NAME: PATISIRAN(ONPATTRO); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/18/1320, 20180829
2937418	19C1002	France		PRODUCT NAME: PATISIRAN; REGISTRATION NO/DATE: EU/1/18/1320 20180829
2937418	2019/004	Ireland		PRODUCT NAME: PATISIRAN; REGISTRATION NO/DATE: EU/1/18/1320 20180827
2937418	3/2019	Austria		PRODUCT NAME: PATISIRAN; REGISTRATION NO/DATE: EU/1/18/1320 (MITTEILUNG) 20180829
2937418	CA 2019 00005	Denmark		PRODUCT NAME: PATISIRAN... (NAVN FOR LANGT); REG. NO/DATE: EU/1/18/1320 20180829

Market Dynamics and Financial Trajectory for ONPATTRO

Introduction

ONPATTRO, developed by Alnylam Pharmaceuticals, is a groundbreaking RNA interference (RNAi) therapeutic approved for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). Since its approval, the drug has shown significant growth and has become a key player in the rare disease treatment market.

Early Launch and Initial Growth

In the first quarter of 2019, ONPATTRO demonstrated strong sales, totaling $26.3 million, more than doubling from the $12.1 million in the fourth quarter of 2018. This growth was driven by an increase in the number of patients on commercial treatment, with over 400 patients by the end of the first quarter, double the number at the end of 2018[1].

Patient Growth and Geographic Expansion

By the end of 2019, over 750 patients worldwide were receiving commercial ONPATTRO, with significant growth seen in the U.S., EU, and Japan. The strong launch was supported by definitive value-based agreements with major commercial payers, covering over 130 million U.S. lives[4].

Revenue Performance

Quarterly and Annual Revenues

• In the fourth quarter of 2019, ONPATTRO achieved global net product revenues of $55.8 million, contributing to a full-year revenue of $166.4 million[4].
• By 2022, ONPATTRO's net product revenues had grown to $137 million for the first quarter, reflecting a steady increase in patient numbers and market penetration[2].
• In the fourth quarter of 2024, ONPATTRO's net product revenues were $79 million, part of a full-year total of $354.5 million[5].

Comparative Growth

The revenue growth of ONPATTRO has been consistent, with a notable increase from $12.5 million in 2018 to $166.5 million in 2019, and further to $354.5 million in 2024. This growth is attributed to the expansion of patient numbers and geographic reach[4][5].

Market Competition

Emergence of Competitors

Despite its strong performance, ONPATTRO faces competition from other treatments for hATTR amyloidosis. Pfizer's tafamidis, approved in Europe, has been a notable competitor. However, Alnylam reported that some patients were being switched from tafamidis to ONPATTRO, indicating physician preference for the RNAi therapeutic[1].

AMVUTTRA as a Successor

In recent years, Alnylam has introduced AMVUTTRA (vutrisiran), another RNAi therapeutic for hATTR amyloidosis. While ONPATTRO's sales have seen a slight decline due to patients switching to AMVUTTRA, the combined revenues of both drugs have contributed to significant growth in the total TTR (transthyretin) segment. In the third quarter of 2024, combined revenues for ONPATTRO and AMVUTTRA were $309 million, representing a 34% annual growth[3].

Physician and Patient Adoption

Increasing Physician Comfort

The adoption of ONPATTRO has been facilitated by increasing physician comfort with the drug. By the first quarter of 2019, prescriptions outside Alnylam's assist program accounted for 20% to 25% of demand, indicating growing confidence among healthcare providers[1].

Patient Switching

There have been instances where patients were switched from other treatments, such as tafamidis, to ONPATTRO, particularly in Europe. This switching trend underscores the therapeutic benefits and patient outcomes associated with ONPATTRO[1].

Financial Guidance and Projections

Revenue Projections

For 2020, Alnylam projected ONPATTRO net product revenues to be between $285 million and $315 million. By 2024, the company updated its guidance, expecting combined net product revenues for ONPATTRO, AMVUTTRA, GIVLAARI, and OXLUMO to be between $1.575 billion and $1.650 billion[2][5].

Operating Expenses

Alnylam has also provided guidance on operating expenses, with GAAP R&D and SG&A expenses projected to be between $2.000 billion and $2.150 billion for 2024. Non-GAAP expenses are expected to be lower, reflecting the exclusion of certain non-cash and non-recurring items[3].

Challenges and Future Outlook

Regulatory and Market Dynamics

Despite the strong growth, Alnylam faces challenges such as regulatory approvals and market competition. The company's ability to maintain its lead will depend on continued innovation, successful clinical trials, and effective market strategies.

Switching to AMVUTTRA

The recent trend of patients switching from ONPATTRO to AMVUTTRA presents both opportunities and challenges. While it indicates a strong product pipeline, it also necessitates strategic management to ensure continued revenue growth from both drugs[3].

Key Takeaways

• Strong Initial Growth: ONPATTRO's sales more than doubled in the first quarter of 2019 compared to the fourth quarter of 2018.
• Geographic Expansion: Significant growth in the U.S., EU, and Japan has contributed to the drug's revenue.
• Competition: ONPATTRO faces competition from tafamidis and internal competition from AMVUTTRA.
• Revenue Projections: Combined revenues for ONPATTRO and AMVUTTRA are expected to continue growing, with projected revenues between $1.575 billion and $1.650 billion for 2024.
• Operational Expenses: Alnylam has provided guidance on GAAP and non-GAAP operating expenses, reflecting the company's financial management strategies.

FAQs

Q: What is ONPATTRO used for?

ONPATTRO is used for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), a rare genetic disorder.

Q: How has ONPATTRO performed financially since its launch?

ONPATTRO has shown significant growth since its launch, with revenues increasing from $12.5 million in 2018 to $354.5 million in 2024.

Q: What are the main competitors for ONPATTRO?

ONPATTRO faces competition from Pfizer's tafamidis and internally from Alnylam's own AMVUTTRA (vutrisiran).

Q: Why are patients switching from ONPATTRO to AMVUTTRA?

Patients are switching due to the therapeutic benefits and potentially better outcomes associated with AMVUTTRA, as well as physician preference.

Q: What are Alnylam's financial projections for ONPATTRO and other products?

Alnylam projects combined net product revenues for ONPATTRO, AMVUTTRA, GIVLAARI, and OXLUMO to be between $1.575 billion and $1.650 billion for 2024.

Sources

1. Biopharma Dive: "Alnylam posts strong Onpattro sales as competition heats up"[1]
2. BioSpace: "Alnylam Pharmaceuticals Reports First Quarter 2022 Financial Results and Highlights Recent Period Activity"[2]
3. BioSpace: "Alnylam Pharmaceuticals Reports Third Quarter 2024 Financial Results and Highlights Recent Period Activity"[3]
4. Alnylam Investors: "Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2019 Financial Results and Highlights Recent Significant Corporate Highlights"[4]
5. Alnylam Investors: "Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2024 Financial Results"[5]