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 fourteen patents protecting this compound. Additional information is available in the individual branded drug profile pages.

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

One supplier is listed for this compound.

Summary for patisiran sodium

International Patents:	251
US Patents:	14
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

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			Y
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			Y
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			Y
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922-001	Aug 10, 2018		RX	Yes	Yes			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
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
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 patisiran sodium

Country	Patent Number	Title	Estimated Expiration
South Korea	101635436
South Africa	201102876	COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TRANSTHYRETIN
Eurasian Patent Organization	201170591
European Patent Office	1765847	ACIDE RIBONUCLEIQUE DOUBLE BRIN RESISTANT AUX NUCLEASES (NUCLEASE RESISTANT DOUBLE-STRANDED RIBONUCLEIC ACID)
Canada	2721333
Eurasian Patent Organization	201792626

Supplementary Protection Certificates for patisiran sodium

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2937418	300965	Netherlands		PRODUCT NAME: PATISIRAN; REGISTRATION NO/DATE: EU/1/18/1320 20180829
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	LUC00098	Luxembourg		PRODUCT NAME: PATISIRAN ET SES DERIVES PHARMACEUTIQUEMENT ACCEPTABLES (ONPATTRO ); AUTHORISATION NUMBER AND DATE: EU/1/18/1320 20180829
2937418	PA2019501	Lithuania		PRODUCT NAME: PATISIRANAS; REGISTRATION NO/DATE: EU/1/18/1320 20180827
2937418	CR 2019 00005	Denmark		PRODUCT NAME: PATISIRAN; REG. NO/DATE: EU/1/18/1320 20180829
2937418	3/2019	Austria		PRODUCT NAME: PATISIRAN; REGISTRATION NO/DATE: EU/1/18/1320 (MITTEILUNG) 20180829

Market Dynamics and Financial Trajectory for Patisiran Sodium

Last updated: July 27, 2025

Introduction

Patisiran sodium, marketed as Onpattro, represents a pioneering therapeutic in the treatment of hereditary transthyretin amyloidosis (hATTR), a rare, life-threatening genetic disorder characterized by amyloid deposits affecting multiple organs. As the first FDA-approved RNA interference (RNAi) therapy, Patisiran has both revolutionized treatment paradigms and shaped the commercial landscape for rare disease therapeutics. Understanding its market dynamics and financial trajectory requires an analysis of competitive factors, regulatory environment, patient demographics, and evolving reimbursement policies.

Market Overview and Size

The global market for hereditary transthyretin amyloidosis (hATTR) is projected to grow substantially, driven by increased disease recognition, improved diagnostic methods, and expanding therapeutic options. Estimates place the prevalence of hATTR at approximately 50,000-100,000 globally, with higher concentrations in Japan, Portugal, Sweden, and the United States (US), where diagnosis rates are improving.

The initial market for Patisiran was limited, given the rarity of the disease; however, recent approvals extending to broader indications and ongoing clinical trials are expected to substantially increase its addressable patient population. According to IQVIA estimates, the global rare disease pharmaceutical market is expected to reach $250 billion by 2030, with RNAi therapies like Patisiran contributing significantly.

Regulatory Milestones and Approvals

Patisiran received FDA approval in August 2018 for the treatment of hATTR amyloidosis with polyneuropathy, a landmark as the first approved RNAi therapeutic [1]. The European Commission followed with approval in 2019. Notably, the FDA granted accelerated approval pathways, reflecting its potential to address untreated patient needs rapidly.

In March 2023, the FDA approved Patisiran for hATTR amyloidosis affecting the cardiomyopathy subset, broadening its market and demonstrating the therapeutic’s evolving role in managing systemic forms of the disease. Regulatory clarity and the approval of companion diagnostics underpin the market's growth potential.

Competitive Landscape

Despite its pioneering status, Patisiran faces competition from alternative therapies, notably Tafamidis (Vyndaqel/Vyndamax), approved in multiple markets for transthyretin amyloid cardiomyopathy and polyneuropathy, and Ionis Pharmaceuticals’ investigational agents exploring next-generation RNAi platforms.

In 2020, Vyndaqel generated global sales exceeding $900 million, indicating strong market traction among clinicians. While Vyndaqel primarily targets transthyretin stabilization rather than RNA interference, its established role creates a competitive environment. Additionally, gene editing approaches and antisense oligonucleotides are emerging as future competitors.

Pricing, Reimbursement, and Access Dynamics

Patisiran’s high treatment cost—reported at approximately $450,000 annually in the US—poses reimbursement challenges but is mitigated by advocacy, rare-disease policies, and value-based arrangements. The price point is characteristic of orphan drugs, which often justify premium pricing through clinical benefits and limited patient populations.

Payers and insurers are increasingly adopting risk-sharing models, including outcomes-based agreements, to manage access risk. The complex administration—intravenous infusion biannually—adds logistical considerations that influence overall treatment affordability and patient adherence.

Market Growth Drivers

Key factors propelling Patisiran’s market expansion include:

• Increased Diagnostic Rates: Improved genetic testing and awareness lead to more diagnosed cases.
• Expanded Indications: Approval for cardiomyopathy broadens patient eligibility.
• Treatment Paradigm Shift: RNAi therapy, with its targeted mechanism, offers an alternative to traditional therapies, possibly leading to earlier intervention.
• Pipeline Developments: Continuous clinical research on next-generation RNAi drugs and combination therapies continues to fuel optimism.

Market Challenges and Risks

Several factors temper optimistic growth projections:

• Pricing and Reimbursement Barriers: High costs may limit access in cost-constrained health systems.
• Therapeutic Competition: Effective, less expensive options like Tafamidis, and potential future therapeutics, threaten market share.
• Limited Awareness and Diagnosis: Many patients remain undiagnosed or misdiagnosed, hampering market penetration.
• Manufacturing and Supply Chain Complexity: As an advanced biologic, Patisiran's production is complex, potentially impacting supply and pricing stability.

Financial Trajectory and Revenue Outlook

Initial breakthrough sales of Patisiran validated its commercial potential. In Q2 2023, Alnylam Pharmaceuticals, the manufacturer, reported $162 million in global product revenues, a significant increase from prior quarters, reflecting market expansion and improved reimbursement [2].

Analysts predict a compound annual growth rate (CAGR) of approximately 15-20% over the next five years, driven by increased diagnosed patient numbers, expanded approvals, and potential new indications. Strategic collaborations with payers and flexibility in pricing models will influence overall revenue trajectories.

Moreover, as data emerges supporting earlier, possibly prophylactic administration, market penetration could accelerate, further elevating revenue streams.

Future Outlook and Market Trends

The future of Patisiran and similar RNAi therapies hinges on several key trends:

• Broader Adoption in Clinical Practice: With growing clinician familiarity, utilization should increase.
• Regulatory Approvals in Additional Markets: Expanding to Asia-Pacific, Latin America, and other regions will diversify revenue sources.
• Advances in Delivery Technologies: Innovations like subcutaneous formulations could ease administration, expanding patient eligibility.
• Expansion to Related Indications: Investigational studies in ATTR-related cardiomyopathies, ocular, and neurological manifestations may generate additional approvals.

Long-term success depends on balancing innovation, reimbursement strategies, and market adaptation in a competitive and rapidly evolving landscape.

Key Takeaways

• Patisiran marked a historic shift in rare disease treatment as the first FDA-approved RNAi therapeutic, with significant market potential driven by rising diagnosis rates and expanding indications.
• The drug is competing primarily with transthyretin stabilizers like Tafamidis, with growth driven by biosimilar and pipeline developments.
• High pricing remains a challenge but is mitigated by rare disease policies and innovative payment models; access and reimbursement strategies will significantly influence market penetration.
• Revenue projections indicate robust growth, with an estimated CAGR of 15-20% over the next five years, contingent upon pipeline success, market access, and healthcare provider adoption.
• Future trends include international market expansion, delivery innovation, and indication broadening, which could further enhance Patisiran’s financial trajectory.

FAQs

1. How does Patisiran differ from other treatments for hereditary transthyretin amyloidosis?
Patisiran employs RNA interference technology to reduce transthyretin protein production, addressing the disease at its molecular root, unlike stabilizers like Tafamidis, which prevent amyloid formation. Its targeted mechanism offers a transformative approach, particularly in neurological manifestations.

2. What are the main challenges in commercializing Patisiran globally?
Challenges include high treatment costs, reimbursement barriers in certain regions, logistical complexities of intravenous administration, and limited awareness among clinicians and patients, especially in regions with underdeveloped genetic testing infrastructure.

3. Are there upcoming therapies that could replace or complement Patisiran?
Yes, investigational agents like Givosiran (another RNAi therapeutic) and NTLA-2001 (a gene-editing therapy) aim to expand treatment options. These could complement or, in some cases, replace Patisiran depending on efficacy, safety, and pricing.

4. What is the potential for Patisiran to treat other amyloidosis subtypes?
Research is ongoing into the utility of Patisiran in cardiac amyloidosis and other systemic forms. Its safety profile and mechanism suggest promising prospects, but regulatory approval will depend on clinical trial outcomes.

5. How might reimbursement models evolve for gene-targeted rare disease therapies like Patisiran?
Expected trends include outcomes-based agreements, annuity or subscription-based models, and tiered pricing strategies aligning cost with clinical value, facilitating broader access and sustainability.

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Sources:

[1] U.S. Food and Drug Administration. FDA Approves Patisiran for Hereditary Transthyretin Amyloidosis. 2018.
[2] Alnylam Pharmaceuticals. Q2 2023 Financial Results.