Vutrisiran sodium - Generic Drug Details

What are the generic 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 thirty-seven patent family members in forty-seven countries.

One supplier is listed for this compound.

Summary for vutrisiran sodium

International Patents:	237
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	9,399,775		Y	Y
Alnylam Pharms Inc	AMVUTTRA	vutrisiran sodium	SOLUTION;SUBCUTANEOUS	215515-001	Jun 13, 2022		RX	Yes	Yes	10,570,391		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	9,370,581		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	11,401,517		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/005852 Treatment 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
Hong Kong	1253410	RNAI試劑、組合物及其用於治療甲狀腺素運載蛋白(TTR)相關疾病的使用方法 (RNAI AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING TRANSTHYRETIN (TTR) ASSOCIATED DISEASES)
Netherlands	301216
South Korea	102095699
Poland	3301177
Israel	296476	תכשירים של טרנסטירתין (ttr) irna ושיטות לשימוש בהם לטיפול או מניעה של מחלות הקשורות ל-ttr (Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated diseases)
Israel	263458	גורמי rnai . תכשירים ושיטות לשימוש בהם לטיפול במחלות הקשורות לטרנסתיראתין (Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases)

Supplementary Protection Certificates for vutrisiran sodium

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3329002	PA2023505	Lithuania		PRODUCT NAME: VUTRISIRANAS; REGISTRATION NO/DATE: EU/1/22/1681 20220915
3329002	C202330008	Spain		PRODUCT NAME: VUTRISIRAN; NATIONAL AUTHORISATION NUMBER: EU/1/22/1681; DATE OF AUTHORISATION: 20220915; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/22/1681; DATE OF FIRST AUTHORISATION IN EEA: 20220915
3329002	122023000011	Germany		PRODUCT NAME: VUTRISIRAN; REGISTRATION NO/DATE: EU/1/22/1681 20220915
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	CA 2023 00008	Denmark		PRODUCT NAME: VUTRISIRAN; REG. NO/DATE: EU/1/22/1681 20220916
3329002	301216	Netherlands		PRODUCT NAME: VUTRISIRAN; REGISTRATION NO/DATE: EU/1/22/1681 20220916

Market Dynamics and Financial Trajectory for Vutrisiran Sodium

Last updated: July 11, 2025

Introduction

Vutrisiran sodium, an innovative RNA interference (RNAi) therapeutic developed by Alnylam Pharmaceuticals, targets hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), a rare but debilitating condition affecting the nervous system and heart. Approved by the FDA in 2022, this drug represents a breakthrough in treating polyneuropathy associated with hATTR amyloidosis, offering patients a subcutaneous injection every three months. As the pharmaceutical industry navigates rising demand for precision medicines, vutrisiran sodium's market dynamics and financial outlook provide critical insights for investors and executives evaluating rare disease therapeutics.

This analysis delves into the competitive landscape, regulatory influences, and revenue projections, highlighting how vutrisiran sodium fits into the broader RNAi and amyloidosis treatment markets. With global healthcare spending on rare diseases projected to grow, understanding these factors helps stakeholders assess investment risks and opportunities in this niche segment.

Overview of Vutrisiran Sodium

Vutrisiran sodium operates by silencing the TTR gene, reducing the production of transthyretin protein and halting amyloid buildup in vital organs. This mechanism distinguishes it from older treatments, delivering superior efficacy in clinical trials, as evidenced by the HELIOS-A study, which showed significant improvements in neuropathy and quality of life metrics.

Launched amid a surge in RNAi therapies, vutrisiran sodium benefits from Alnylam's patent portfolio, which extends exclusivity through 2037 in key markets. This protection shields it from immediate generic threats, allowing Alnylam to command premium pricing—around $24,000 per vial in the U.S., adjusted for patient assistance programs. However, pricing pressures from payers and international regulators could influence long-term adoption, particularly in Europe where health technology assessments scrutinize cost-effectiveness.

The drug's market entry aligns with a growing patient pool; estimates suggest 50,000 individuals worldwide live with hATTR amyloidosis, with diagnosis rates improving due to advanced genetic testing. Alnylam's strategic partnerships, such as with Regeneron for RNAi platform expansion, bolster its supply chain and distribution, ensuring steady market penetration.

Current Market Dynamics

The hATTR amyloidosis market, valued at approximately $1.5 billion in 2023, is expanding rapidly, driven by an aging population and better disease awareness [1]. Vutrisiran sodium captures a significant share, competing directly with Pfizer's tafamidis, which dominates with annual sales exceeding $1 billion. While tafamidis offers oral convenience, vutrisiran's tri-monthly dosing and clinical edge in neuropathy reversal position it as a preferred option for severe cases.

Regulatory milestones shape these dynamics; the EMA granted conditional approval in 2022, facilitating European access, but ongoing post-marketing studies could impose additional requirements. In the U.S., the FDA's orphan drug designation grants seven years of market exclusivity, enhancing vutrisiran's competitive barrier. However, emerging biosimilars and pipeline drugs from Ionis Pharmaceuticals threaten to erode this advantage by mid-decade.

Demand fluctuates by region: North America leads with 40% of global sales, fueled by robust reimbursement and early adopter prescribers, while Asia-Pacific regions lag due to diagnostic gaps and economic constraints. Supply chain disruptions, highlighted during the COVID-19 era, add volatility; Alnylam mitigated this by investing in diversified manufacturing, including facilities in Europe.

Market consolidation among pharmaceutical giants further intensifies competition. For instance, Novartis's acquisition of AveXis expanded its gene therapy capabilities, indirectly pressuring RNAi players like Alnylam to innovate faster. Analysts forecast the overall RNAi market to reach $5 billion by 2028, with vutrisiran sodium contributing substantially as a flagship product [2].

Financial Performance and Projections

Alnylam's financial trajectory for vutrisiran sodium reflects strong early performance, with 2023 revenues hitting $280 million, a 150% year-over-year increase. This growth stems from expanded market access and rising prescriptions, projecting to surpass $1 billion annually by 2026 as global approvals broaden [3]. The drug's gross margin exceeds 80%, driven by efficient production and high pricing power in orphan drug markets.

R&D investments, totaling $500 million in 2023, underpin this trajectory; Alnylam allocates resources to expand vutrisiran's indications, including cardiac amyloidosis, potentially doubling its addressable market. Partnerships, such as the $800 million deal with Roche for RNAi co-development, provide non-dilutive funding, easing cash flow pressures.

However, financial risks loom. Patent expirations post-2037 could invite generics, slashing prices by 50-70%. Current stock valuations reflect optimism, with Alnylam's shares up 30% in 2024, but macroeconomic factors like inflation and currency fluctuations impact international sales. In Europe, where pricing negotiations with bodies like NICE in the UK have delayed reimbursement, revenues trail U.S. figures by 20%.

Projections from equity research firms indicate a compound annual growth rate (CAGR) of 25% for vutrisiran sodium through 2030, contingent on successful trial outcomes and minimal regulatory hurdles. Investors should monitor cash reserves, as Alnylam burned through $200 million in operating expenses last quarter, emphasizing the need for sustained revenue growth to fund innovation.

Challenges and Opportunities

Vutrisiran sodium faces hurdles in a fragmented regulatory environment; varying approval timelines across countries delay global rollout, potentially capping short-term revenues. Payer scrutiny over high costs—exacerbated by the Inflation Reduction Act in the U.S.—may force price concessions, impacting profitability.

Conversely, opportunities abound in untapped markets. Emerging economies in Latin America and Asia offer growth potential, with Alnylam eyeing partnerships to navigate local barriers. The drug's safety profile, with low discontinuation rates in trials, enhances patient retention and long-term revenue stability.

Sustainability initiatives, such as Alnylam's focus on eco-friendly manufacturing, could differentiate it in an ESG-conscious investment landscape. Additionally, synergistic combinations with other therapies, like beta-blockers for cardiac patients, might expand its utility and drive cross-selling.

Conclusion

Vutrisiran sodium stands as a pivotal asset in Alnylam's portfolio, navigating complex market dynamics to deliver substantial financial returns. Its role in advancing rare disease treatments underscores the pharmaceutical sector's shift toward targeted therapies, where innovation meets commercial viability.

Key Takeaways

• Vutrisiran sodium's market share in hATTR amyloidosis grows steadily, outpacing competitors like tafamidis through superior efficacy and dosing convenience.
• Financial projections forecast revenues exceeding $1 billion by 2026, supported by patent protections and strategic alliances, though regulatory and pricing challenges pose risks.
• Investors should prioritize monitoring R&D expansions and global reimbursement trends to gauge long-term viability in the evolving RNAi landscape.
• The drug's success highlights the importance of orphan drug designations in driving profitability amid rising healthcare costs.
• Opportunities in emerging markets and new indications could offset potential patent-related threats, positioning Alnylam for sustained growth.

FAQs

1. What makes vutrisiran sodium different from other hATTR amyloidosis treatments?
Vutrisiran sodium uses RNAi technology to specifically silence the TTR gene, offering faster neuropathy improvements compared to tafamidis, which only stabilizes the protein without reducing its production.

2. How does pricing affect vutrisiran sodium's market access?
At around $24,000 per vial, the drug faces reimbursement hurdles in regions with strict cost controls, but patient assistance programs in the U.S. help mitigate access issues for eligible patients.

3. What are the main financial risks for investors in vutrisiran sodium?
Key risks include patent expiration after 2037, potential generic competition, and regulatory delays that could slow revenue growth in international markets.

4. How has Alnylam expanded vutrisiran sodium's potential applications?
Alnylam is investigating its use in cardiac amyloidosis through ongoing trials, which could broaden the patient base and enhance revenue streams beyond the current polyneuropathy focus.

5. What role do partnerships play in vutrisiran sodium's financial trajectory?
Collaborations, such as with Roche, provide funding and expertise for R&D, reducing Alnylam's financial burden and accelerating market expansion efforts.

Sources

1. Alnylam Pharmaceuticals. Annual Report 2023. Retrieved from Alnylam investor relations page.
2. Grand View Research. RNAi Therapeutics Market Analysis Report, 2024.
3. Evaluate Pharma. World Preview 2024, Outlook to 2030.