VUTRISIRAN SODIUM - Generic Drug Details

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

Vutrisiran 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.

Vutrisiran sodium has two hundred and fifty-five patent family members in forty-eight countries.

One supplier is listed for this compound.

Summary for VUTRISIRAN SODIUM

International Patents:	255
US Patents:	13
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
DailyMed Link:	VUTRISIRAN SODIUM at DailyMed

Pharmacology for VUTRISIRAN SODIUM

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

Anatomical Therapeutic Chemical (ATC) Classes for VUTRISIRAN 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 VUTRISIRAN 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	AMVUTTRA	vutrisiran sodium	SOLUTION;SUBCUTANEOUS	215515-001	Jun 13, 2022		RX	Yes	Yes	8,106,022		Y	Y
Alnylam Pharms Inc	AMVUTTRA	vutrisiran sodium	SOLUTION;SUBCUTANEOUS	215515-001	Jun 13, 2022		RX	Yes	Yes	10,612,024		Y	Y
Alnylam Pharms Inc	AMVUTTRA	vutrisiran sodium	SOLUTION;SUBCUTANEOUS	215515-001	Jun 13, 2022		RX	Yes	Yes	10,131,907		Y	Y
Alnylam Pharms Inc	AMVUTTRA	vutrisiran sodium	SOLUTION;SUBCUTANEOUS	215515-001	Jun 13, 2022		RX	Yes	Yes	8,828,956		Y	Y
Alnylam Pharms Inc	AMVUTTRA	vutrisiran sodium	SOLUTION;SUBCUTANEOUS	215515-001	Jun 13, 2022		RX	Yes	Yes	10,208,307		Y	Y
Alnylam Pharms Inc	AMVUTTRA	vutrisiran sodium	SOLUTION;SUBCUTANEOUS	215515-001	Jun 13, 2022		RX	Yes	Yes	9,370,581		Y	Y
Alnylam Pharms Inc	AMVUTTRA	vutrisiran sodium	SOLUTION;SUBCUTANEOUS	215515-001	Jun 13, 2022		RX	Yes	Yes	9,399,775		Y	Y

EU/EMA Drug Approvals for VUTRISIRAN SODIUM

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Alnylam Netherlands B.V.	Amvuttra	vutrisiran	EMEA/H/C/005852Treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.	Authorised	no	no	yes	2022-09-15

International Patents for VUTRISIRAN SODIUM

Country	Patent Number	Title	Estimated Expiration
European Patent Office	3705125	CONJUGUÉS GLUCIDIQUES UTILISÉS EN TANT QU'AGENTS D'ADMINISTRATION POUR DES OLIGONUCLÉOTIDES (CARBOHYDRATE CONJUGATES AS DELIVERY AGENTS FOR OLIGONUCLEOTIDES)
Eurasian Patent Organization	201790420	СРЕДСТВА, ПРЕДСТАВЛЯЮЩИЕ СОБОЙ МОДИФИЦИРОВАННУЮ ДВУХНИТЕВУЮ РНК
Malaysia	195720
Japan	2017002082	標的化脂質 (TARGETED LIPID)
Hong Kong	1200191
Mexico	2017002144	AGENTES DE ÁCIDO RIBONUCLEICO (ARN) DE CADENA DOBLE MODIFICADOS. (MODIFIED DOUBLE-STRANDED RNA AGENTS.)
Canada	2910760

Supplementary Protection Certificates for VUTRISIRAN SODIUM

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3329002	C20230004 00401	Estonia		PRODUCT NAME: VUTRISIRAAN;REG NO/DATE: EU/1/22/1681 16.09.2022
3329002	C03329002/01	Switzerland		PRODUCT NAME: VUTRISIRAN; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 69074 23.06.2023
3329002	122023000011	Germany		PRODUCT NAME: VUTRISIRAN; REGISTRATION NO/DATE: EU/1/22/1681 20220915
3329002	CA 2023 00008	Denmark		PRODUCT NAME: VUTRISIRAN; REG. NO/DATE: EU/1/22/1681 20220916
3329002	2390008-7	Sweden		PRODUCT NAME: VUTRISIRAN; REG. NO/DATE: EU/1/22/1681 20220916
3329002	LUC00297	Luxembourg		PRODUCT NAME: VUTRISIRAN SOUS TOUTES LES FORMES PROTEGEES PAR LE BREVET DE BASE; AUTHORISATION NUMBER AND DATE: EU/1/22/1681 20220916
3329002	PA2023505	Lithuania		PRODUCT NAME: VUTRISIRANAS; REGISTRATION NO/DATE: EU/1/22/1681 20220915

Market Dynamics and Financial Trajectory for Vutrisiran Sodium

Last updated: July 27, 2025

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Introduction

Vutrisiran sodium represents a significant advancement in RNA interference (RNAi) therapeutics, targeting transthyretin-mediated amyloidosis (ATTR). As a subcutaneously administered siRNA drug, vutrisiran offers promising clinical benefits for patients with hereditary transthyretin amyloidosis (hATTR), including cardiomyopathy and polyneuropathy. This analysis examines the current market landscape, competitive positioning, regulatory considerations, and financial prospects shaping the trajectory of vutrisiran sodium over the coming years.

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Therapeutic Landscape and Market Drivers

The ATTR market has historically depended on limited treatment options, with liver transplant and TTR-stabilizers like tafamidis serving as primary approaches. The advent of RNAi-based therapies—including vutrisiran—introduces a paradigm shift by directly silencing pathological TTR production.

Key market drivers include:

• Unmet Medical Needs: No cure exists; patients suffer progressive, life-threatening symptoms. Vutrisiran aims to fill this gap with more effective symptom control and disease modification.

• Regulatory Approvals: In August 2022, the U.S. Food and Drug Administration (FDA) granted vutrisiran accelerated approval for hereditary ATTR polyneuropathy (hATTR-PN). Such regulatory progress accelerates market access and confidence.

• Advances in RNAi Therapeutics: The success of similar drugs like patisiran (Onpattro) exemplifies the viability of RNAi platforms, reducing developmental risk.

• Growing Awareness and Diagnostic Capabilities: Increasing recognition, improved disease screening, and genetic testing broaden potential patient pools.

• Pricing and Reimbursement Dynamics: High-value pricing models characteristic of rare disease treatments sustain revenue streams, contingent on reimbursement negotiations.

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Market Penetration and Competitive Positioning

Current Competitors:

• Patisiran (Onpattro): The first FDA-approved RNAi therapy for hATTR-PN, marketed by Alnylam Pharmaceuticals, with established clinical data and market presence.

• TTR-stabilizers (Tafamidis): Market leader for transthyretin amyloid cardiomyopathy and polyneuropathy, with pivotal approvals in multiple jurisdictions.

• Other Emerging Candidates: Companies like Akcea and Ionis are developing related antisense oligonucleotides, while gene-editing approaches remain at early stages.

Vutrisiran’s Differentiators:

• Enhanced Efficacy: Clinical trials suggest improved TTR knockdown and potentially better symptom control over existing therapies.

• Convenient Dosing: Once every six months injections may improve patient compliance compared to other treatments requiring more frequent dosing.

• Favorable Safety Profile: Preliminary data indicates tolerability comparable to or better than competitors, reducing barriers to uptake.

Market Penetration Strategy:

• Targeting Specialist Centers: Rheumatology and cardiology clinics specialized in amyloidosis remain primary channels.

• Market Access and Reimbursement: Aligning with payers through health economic assessments will determine pricing and reimbursement prospects.

• Expanding Indications: Beyond hATTR, potential exists in wild-type ATTR cardiomyopathy, broadening the addressable market.

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Regulatory and Patent Landscape

Vutrisiran’s accelerated approval signifies robust regulatory support, yet full approval processes and post-marketing commitments remain in progress. Patent exclusivity protection for the siRNA platform and specific formulation rights underpin future revenue streams, although the crowded R&D pipeline necessitates proactive patent strategies.

Regulatory hurdles and post-approval obligations could impact timelines and revenue projections. The expanding global regulatory landscape, including European Medicines Agency (EMA) approvals, will influence market scope.

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Financial Projections and Market Potential

Market Size Estimates:

The global hereditary ATTR amyloidosis market was valued at approximately USD 500 million in 2022, with forecasts exceeding USD 2 billion by 2030, driven by increased diagnostics and expanding indications (source: FDA and industry reports).

Revenue Outlook:

• Initial Adoption Period: Limited by clinical adoption curves, early revenues likely in the hundreds of millions annually within the first three years post-approval.

• Growth Factors:

  • Increased diagnostic rates and awareness.

  • Broadened indications, including wild-type ATTR.

  • Competitive differentiation through dosing convenience and efficacy.

• Risks and Challenges:

  • Market penetration may be hampered by high treatment costs and payer hesitancy.

  • Competition from existing therapies may restrict market share gains.

  • Potential clinical development delays or adverse safety reports could temper growth.

Financial Trajectory:

Based on comparable rare disease therapeutics, vutrisiran’s revenues could reach USD 500 million to USD 1 billion by 2028, assuming successful market access, pricing, and adoption. Margins are expected to improve with manufacturing scale and optimized supply chains, supporting sustained profitability.

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Strategic Opportunities and Challenges

Opportunities:

• Pipeline Expansion: Developing formulations or combination therapies targeting other amyloidosis subtypes.

• Global Expansion: Launches in Asian and European markets to capitalize on unmet needs.

• Data Generation: Demonstrating long-term efficacy and safety could lead to expanded indications and premium pricing.

Challenges:

• Pricing Pressures: Payer pushback on high-cost therapies may necessitate innovative reimbursement strategies.

• Competitive Dynamics: A crowded pipeline requires strategic differentiation and robust clinical data.

• Manufacturing Complexity: RNAi therapeutics entail complex production, affecting scalability and costs.

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Conclusion

Vutrisiran sodium is poised for a transformative role in treating hereditary transthyretin amyloidosis, backed by regulatory support and promising clinical data. Its market success hinges on clinical efficacy, payer acceptance, and strategic market access. While challenges persist, the drug’s innovative profile and expanding indications position it favorably within a growing rare disease therapeutics market, with substantial financial potential over the upcoming decade.

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Key Takeaways

• Vutrisiran’s RNAi mechanism offers a targeted and potentially superior treatment for ATTR, with advantages in dosing and efficacy.

• The market for ATTR therapeutics is expanding, driven by increased diagnosis and unmet needs, positioning vutrisiran favorably.

• Competitive differentiation, regulatory strategy, and payer engagement will be critical in realizing its financial potential.

• Revenue forecasts suggest the global market could surpass USD 2 billion by 2030, with significant upside from expanded indications.

• Strategic execution and robust clinical data will determine its market share and long-term profitability.

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FAQs

1. What sets vutrisiran sodium apart from existing ATTR treatments?
Vutrisiran offers a twice-yearly subcutaneous dose, potentially improving adherence over less convenient alternatives like patisiran and tafamidis. Its mechanism—RNA interference—also promises more profound TTR suppression, potentially leading to better clinical outcomes.

2. What are the main regulatory milestones for vutrisiran?
Following FDA accelerated approval in August 2022 for hereditary ATTR polyneuropathy, subsequent milestones include full approval, EMA licensing, and additional indications approval, which will enhance market penetration.

3. How does the competitive landscape impact vutrisiran’s growth?
While patisiran and tafamidis dominate the current market, vutrisiran’s differentiated dosing and efficacy may provide a competitive edge. However, high drug costs and payer skepticism pose hurdles to rapid adoption.

4. What is the projected revenue potential for vutrisiran?
Industry estimates project revenues between USD 500 million and USD 1 billion annually by 2028, contingent upon approval, market access, and clinical success across multiple regions.

5. What are the main risks influencing vutrisiran’s financial trajectory?
Risks include delayed regulatory approvals, manufacturing complexities, high treatment costs leading to reimbursement challenges, and intensifying competition within the RNAi and broader amyloidosis treatment markets.

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Sources:

[1] FDA Press Release, 2022. "FDA Grants Accelerated Approval to Vutrisiran for Hereditary ATTR Amyloidosis."
[2] Industry Reports on Rare Disease Market, 2023.
[3] Alnylam Pharmaceuticals, Vutrisiran Clinical Data Reports, 2022–2023.
[4] Market Research on ATTR Amyloidosis Treatments, 2023.
[5] Regulatory Agency Guidelines on Rare Disease Therapies, 2023.