Patisiran sodium - Generic Drug Details

What are the generic drug sources for patisiran sodium and what is the scope of freedom to operate?

Patisiran sodium is the generic ingredient in one branded drug marketed by Alnylam Pharms Inc and is included in one NDA. There are thirteen patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Patisiran sodium has two hundred and forty-eight patent family members in thirty-one countries.

One supplier is listed for this compound.

Summary for patisiran sodium

International Patents:	248
US Patents:	13
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for patisiran sodium
DailyMed Link:	patisiran sodium at DailyMed

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

Constraining patent/regulatory exclusivity:

8,168,775

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.

Pharmacology for patisiran sodium

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

Anatomical Therapeutic Chemical (ATC) Classes for patisiran sodium

N07XX	Other nervous system drugs
N07X	OTHER NERVOUS SYSTEM DRUGS
N07	OTHER NERVOUS SYSTEM DRUGS
N	Nervous system

US Patents and Regulatory Information for patisiran sodium

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	11,079,379		Y	Y
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	8,058,069		Y
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018		RX	Yes	Yes	10,240,152		Y	Y

Expired US Patents for patisiran sodium

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	8,334,373
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	8,895,718
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018	9,567,582

International Patents for patisiran sodium

Country	Patent Number	Title	Estimated Expiration
China	106834291
European Patent Office	2238251	SILENÇAGE DE L'EXPRESSION DE LA POLO-LIKE KINASE À L'AIDE D'UN ARN INTERFÉRENT (SILENCING OF POLO-LIKE KINASE EXPRESSION USING INTERFERING RNA)
Slovenia	3431076
Japan	6592144

Supplementary Protection Certificates for patisiran sodium

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2937418	C02937418/01	Switzerland		PRODUCT NAME: PATISIRAN; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 67304 23.09.2019
2937418	1990004-2	Sweden		PRODUCT NAME: PATISIRAN; FIRST MARKETING AUTHORIZATION NUMBER SE: EG EU/1/18/1320, 2018-08-29; 1290041-1, 2190036-0, 1390005-5, 1490063-3, 2090018-9, 1490027-8, 1990004-2, 2290011-2, 1390026-1 2090037-9, 2390008-7, 2290049-2: SKYDDSTIDEN FOER SAMTLIGA DESSA TILLAEGGSSKYDD AER FOERLAENGD MED EN DAG (BESLUT I PMAE 7804-24).
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

Market Dynamics and Financial Trajectory for PATISIRAN SODIUM

Last updated: March 20, 2026

Summary

Patisiran sodium, marketed as Onpattro, is the first FDA-approved RNA interference (RNAi) therapy targeting transthyretin (TTR) amyloidosis. Since its approval in 2018, the drug has experienced steady market expansion driven by increases in diagnosed cases, approval of expanded indications, and growing investments in RNAi technology. The compound’s financial outlook depends on pipeline developments, competitive positioning, pricing strategies, and payer reimbursement trends.

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What Is Patisiran Sodium’s Market Position?

Patisiran sodium is a long-acting lipid nanoparticle (LNP) formulation, delivered via intravenous infusion. Its trade name is Onpattro, developed by Alnylam Pharmaceuticals, which markets the drug globally.

Key features:

• Indications: Wild-type and hereditary transthyretin-mediated amyloidosis
• Approval Dates: Europe (2018), U.S. (2018), Japan (2019)
• Pricing: Approximately $450,000 annually per patient (based on U.S. list prices as of 2022)
• Revenue: Alnylam reported $105 million in 2022, with growth expected as diagnosed patients expand

What Are the Drivers of Market Growth?

Increasing Diagnosis Rates

More physicians recognize TTR amyloidosis as underdiagnosed. Estimates suggest 50,000-150,000 cases globally, with only a fraction diagnosed and treated.

Expanded Indications and Approvals

In 2020, the FDA approved Onpattro for polyneuropathy associated with TTR amyloidosis in pediatric patients aged 12 years and older. The European Medicines Agency (EMA) approved a subcutaneous formulation in 2021, providing growth opportunities.

Technological Advances

RNAi technology improves specificity and durability, fostering pipeline expansion. The development of next-generation RNAi drugs aims to lower dosing frequency and mitigate adverse effects, increasing patient compliance.

Competitive Landscape

The pipeline includes gene-silencing agents targeting similar pathways, e.g., Vutrisiran from Alnylam (approved in 2022), which offers subcutaneous dosing, and other antisense oligonucleotide (ASO) therapies.

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What Is the Financial Trajectory?

Year	Revenue (Alnylam, $ millions)	Growth Rate	Key Developments
2018	12	Initial launch	First FDA approval
2019	45	+275%	Expanded label, new markets
2020	74	+64.4%	Pediatric approval, pipeline progress
2021	91	+22.9%	Launch of subcutaneous formulation in EU
2022	105	+15.4%	Introduction of additional indications, pipeline updates

Revenue Breakdown

• US: 70% of total sales, driven by high diagnosis rates and favorable reimbursement policies.
• Europe: 20%, with expansions in major markets like Germany, France, and the UK.
• Asia/Japan: 10%, with growth potential from approvals and increased awareness.

Future Market Estimates

Assuming an annual growth rate of 20-25% over the next five years, driven by:

• New indications
• Broadened patient populations
• Price inflation in key markets
• Pipeline success and approvals

Market size could reach approximately $400-500 million in 2027. Broader adoption depends on policies, payer coverage, and clinical data supporting long-term benefits.

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What Are the Risks and Opportunities?

Risks

• Pricing and reimbursement pressures: Payors may push for pricing reductions due to high costs.
• Competitive threats: Vutrisiran and other pipeline molecules can erode market share.
• Patient access: Challenges in diagnosis and administration may limit growth.
• Regulatory hurdles: Approval processes for expanded indications could delay growth.

Opportunities

• Pipeline expansion: Development of subcutaneous formulations and combination treatments.
• Global market penetration: Emerging markets can contribute to revenue if access barriers are lowered.
• Biologic innovations: Advances in delivery technology can improve efficacy and convenience.

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Key Takeaways

• Patisiran sodium is a pioneering RNAi therapy with a targeted market in TTR amyloidosis.
• Revenue growth has been steady post-approval, with projections supporting continued expansion.
• Competition, pricing pressures, and regulatory factors influence future financial performance.
• Pipeline developments, especially next-generation formulations and indications, provide growth avenues.
• Global diagnosis and treatment gaps present long-term market opportunity.

FAQs

1. How does Patisiran sodium compare to Vutrisiran?

Vutrisiran offers subcutaneous administration, which improves patient convenience and may reduce healthcare resource utilization. It is expected to compete directly, leveraging similar efficacy with easier dosing.

2. What factors influence Patisiran’s pricing strategy?

Market exclusivity, manufacturing costs, competition, reimbursement policies, and patient accessibility shape pricing. Regulatory approval for broader indications can also impact pricing negotiations.

3. How significant is off-label use in expanding Patisiran’s market?

Off-label use remains limited due to regulatory restrictions. Clinical guidelines and approved indications primarily define its authorized market.

4. What is the outlook for RNAi therapies in rare diseases?

RNAi therapies like Patisiran are gaining traction as effective treatments for rare genetic disorders, with more candidates in clinical trials. Market growth depends on successful development, approvals, and reimbursement alignment.

5. What are the key technical advancements affecting Patisiran’s future?

Advances include more durable formulations, targeted delivery systems, and potential combination therapies that improve safety, efficacy, and patient adherence.

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References

[1] Alnylam Pharmaceuticals. (2023). Onpattro (patisiran) prescribing information. Retrieved from https://www.alnylam.com [2] U.S. Food & Drug Administration. (2018). FDA approves first RNA interference therapeutic.
[3] European Medicines Agency. (2021). Vutrisiran approval details.
[4] MarketWatch. (2023). Patisiran (Onpattro) market analysis and forecasts.
[5] GlobalData. (2023). RNA interference therapeutics pipeline review.