Nusinersen sodium - Generic Drug Details

What are the generic sources for nusinersen sodium and what is the scope of patent protection?

Nusinersen sodium is the generic ingredient in one branded drug marketed by Biogen Idec and is included in one NDA. There are nine patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Nusinersen sodium has one hundred and five patent family members in twenty-eight countries.

One supplier is listed for this compound.

Summary for nusinersen sodium

International Patents:	105
US Patents:	9
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for nusinersen sodium
DailyMed Link:	nusinersen sodium at DailyMed

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

Dosage:

SOLUTION;INTRATHECAL

^*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 nusinersen sodium

Drug Class	Antisense Oligonucleotide Survival Motor Neuron-2-directed RNA Interaction
Physiological Effect	Increased Protein Synthesis

Anatomical Therapeutic Chemical (ATC) Classes for nusinersen sodium

M09AX	Other drugs for disorders of the musculo-skeletal system
M09A	OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
M09	OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
M	Musculo-skeletal system

US Patents and Regulatory Information for nusinersen sodium

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016		RX	Yes	Yes				Y
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016		RX	Yes	Yes
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016		RX	Yes	Yes			Y	Y
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016		RX	Yes	Yes
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016		RX	Yes	Yes
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016		RX	Yes	Yes

Expired US Patents for nusinersen sodium

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016

International Patents for nusinersen sodium

Country	Patent Number	Title	Estimated Expiration
Cyprus	1120807
China	115227709
Slovenia	3449926
Russian Federation	2015139553
Portugal	3449926
Cyprus	2017040

Supplementary Protection Certificates for nusinersen sodium

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2548560	2017C/048	Belgium		PRODUCT NAME: SPINRAZA - NUSINERSEN; AUTHORISATION NUMBER AND DATE: EU/1/17/1188 20170601
2548560	57/2017	Austria		PRODUCT NAME: NUSINERSEN UND DESSEN SALZE; REGISTRATION NO/DATE: EU/1/17/1188 MITTEILUNG 20170601
2548560	C 2017 045	Romania		PRODUCT NAME: NUSINERSEN SAU SARURI ALE ACESTUIA; NATIONAL AUTHORISATION NUMBER: EU/1/17/1188; DATE OF NATIONAL AUTHORISATION: 20170530; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/17/1188; DATE OF FIRST AUTHORISATION IN EEA: 20170530
1910395	2017/051	Ireland		PRODUCT NAME: NUSINERSEN OR SALTS THEREOF; REGISTRATION NO/DATE: EU/1/17/1188 20170530
2548560	797	Finland
2548560	C20170038 00228	Estonia		PRODUCT NAME: NUSINERSEEN;REG NO/DATE: EU/1/17/1188 01.06.2017

Market Dynamics and Financial Trajectory for Nusinersen Sodium

Last updated: September 30, 2025

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Introduction

Nusinersen sodium, marketed as Spinraza, is a groundbreaking therapeutic developed for the treatment of spinal muscular atrophy (SMA). Since its FDA approval in 2016, it has significantly altered the landscape of neuromuscular disease management. This analysis explores the evolving market dynamics and financial trajectory of nusinersen sodium, examining factors driving demand, competitive positioning, regulatory developments, and financial performance within the emerging landscape of antisense oligonucleotide therapies.

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Market Overview and Therapeutic Significance

Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder characterized by degeneration of motor neurons, leading to progressive muscle weakness and atrophy. Prior to nusinersen, treatment options were limited, primarily supportive care. The approval of Spinraza by the FDA catalyzed a paradigm shift, establishing the therapy as a first-in-class treatment targeting SMN2 gene splicing to increase production of functional SMN protein.

The market for SMA therapeutics expanded rapidly, driven by both the high unmet medical need and the significant clinical efficacy demonstrated in trials. Nusinersen’s pioneering status set a precedent for antisense oligonucleotide (ASO) therapeutics, fostering an ecosystem that incentivizes innovation and investment.

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Market Dynamics

1. Growing Incidence and Market Penetration

The global incidence of SMA is estimated at approximately 1 in 10,000 live births, with carrier frequencies around 1 in 50 [1]. The large pediatric patient population and the increasing recognition of SMA's broader spectrum, including later-onset forms, have expanded market potential. Initial uptake was slow due to high costs and logistical challenges of intrathecal administration, but subsequent years saw accelerated adoption aided by positive clinical outcomes and expanding indications.

2. Price Accessibility and Reimbursement Landscape

Nusinersen's pricing, set at approximately $750,000 for the initial year and $375,000 annually thereafter in the US, has been a central factor shaping market dynamics. While the high price underscores the therapy's innovative value, it also invites scrutiny over affordability and reimbursement negotiations. Payer willingness is influenced by demonstrable long-term benefits, including improved survival rates and reduced healthcare costs owing to decreased supportive care needs.

The development of alternative therapies, including gene replacements and small molecules, further complicates pricing and reimbursement strategies. Payers increasingly demand comprehensive value assessments, including quality-adjusted life years (QALYs) and real-world effectiveness data.

3. Competitive Landscape and Pipeline Progression

Nusinersen faced competition from emerging SMA treatments, notably:

• Zolgensma (AVXS-101): A gene therapy approved by FDA in 2019, offering a potentially curative single-dose option.
• Risdiplam (Evrysdi): An oral SMN2 splicing modifier, approved in 2020, offering ease of administration and expanded access for various age groups.

The competition has shifted the market toward a multi-therapeutic segment with diverse administration routes, affecting nusinersen’s market share. Nonetheless, established efficacy data and ongoing clinical trials sustain its relevance.

4. Regulatory and Geographic Expansion

Regulatory approvals have expanded beyond the US and EU to Japan, Canada, and other markets, broadening patient access. The approval process in emerging markets remains complex due to cost and regulatory variability, but ongoing negotiations aim to improve access pathways.

Additional licensing and market penetration are contingent on regional reimbursement agreements and healthcare infrastructure capacity for intrathecal injections, especially in resource-limited settings.

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Financial Trajectory Analysis

1. Revenue and Sales Patterns

Since launch, Spinraza has generated billions of dollars globally. In 2021, Biogen reported approximately $1.5 billion in Spinraza sales [2], indicating sustained high demand. The initial peak capitalized on the novelty; however, sales growth plateaued as competition increased and off-label use moderated.

2. Impact of Competition and New Approvals

The market share of nusinersen has seen erosion, especially after evo-tionary approvals of oral risdiplam and gene therapy Zolgensma. While Zolgensma's uniform single-dose regimen appeals for its convenience, its high upfront cost coupled with limited age applicability affects broader utilization.

Risdiplam’s oral administration complements nusinersen, especially in older patients, and offers a more cost-effective alternative, impacting nusinersen's market share. Nonetheless, long-term comparative studies on efficacy and durability remain ongoing.

3. Cost-Effectiveness and Reimbursement Policies

Cost-effectiveness assessments increasingly influence financial trajectories. Payers frequently emphasize value-based agreements, which may include usage caps, outcomes-linked rebates, or managed access schemes to manage financial risk [3].

Biogen's strategic responses include negotiations for favorable reimbursement terms, pricing strategies, and expanding indications, such as pre-symptomatic treatment in infants identified via newborn screening programs.

4. R&D Investment and pipeline prospects

Investments in research exploring combination therapies, gene editing, and alternative delivery routes aim to enhance therapeutic efficacy and address unmet needs. These pipeline developments could reshape the financial outlook by introducing next-generation SMA treatments or improving existing therapies.

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Future Market Trajectory

1. Emerging Therapeutic Options and Market Share Redistribution

The therapeutic landscape is poised for further transformation as gene therapy, RNA editing, and protein replacement strategies mature. The competitive advantage of nusinersen hinges on its proven efficacy, established safety profile, and existing healthcare infrastructure. However, innovations could threaten its market dominance.

2. Regulatory and Policy Shifts

Global adoption will increasingly depend on health policy evolution, with emphasis on early diagnosis via newborn screening, and value-based pricing models. Regulatory agencies may demand more robust long-term data, influencing product positioning and market longevity.

3. Pricing and Reimbursement Trends

Price reductions and innovative reimbursement schemes will likely become more prevalent, driven by healthcare cost containment imperatives. Manufacturer willingness to adapt pricing to regional economic contexts will be crucial for sustained market access.

4. Long-term Financial Outlook

Projected revenue streams of nusinersen are expected to plateau or decline by the late 2020s as competition intensifies and alternative therapies gain ground. However, the drug's established position and ongoing pipeline development offer avenues for sustained revenue through indications expansion and combination therapies.

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Key Takeaways

• Rapid Market Growth: Since its 2016 approval, nusinersen has achieved significant revenue, driven by high unmet need and demonstrated clinical efficacy.
• Competitive Challenges: The arrival of gene therapy (Zolgensma) and oral Risdiplam diversifies treatment options, pressuring nusinersen’s market share.
• Pricing and Reimbursement Pressures: High therapy costs necessitate innovative reimbursement models, influencing profit margins and adoption.
• Pipeline and Innovation: Ongoing research into next-generation therapies may extend nusinersen’s relevance, but also risks obsolescence if superior options emerge.
• Global Accessibility: Geographic expansion and policy shifts toward early diagnosis and value-based care are critical to future growth.
• Financial Outlook: While revenues remain substantial in the near term, long-term growth depends on strategic positioning amid evolving competitive and regulatory environments.

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FAQs

1. How does nusinersen differ from other SMA treatments?
Nusinersen is an antisense oligonucleotide administered via intrathecal injection, targeting SMN2 splicing to increase SMN protein. In contrast, Zolgensma is a gene therapy offering a one-time curative approach, and Risdiplam is an oral medication modifying SMN2 splicing systemically.

2. What are the primary financial challenges facing nusinersen’s market sustainability?
High pricing, reimbursement complexities, emerging competition, and evolving regulatory requirements pose challenges to maintaining and expanding revenue streams.

3. How has the COVID-19 pandemic affected nusinersen’s market dynamics?
Disruptions in healthcare services and patient access temporarily impacted administration rates, but the therapy's essential nature led to resilient demand as health systems adapted.

4. What role does newborn screening play in nusinersen’s future market?
Early diagnosis via newborn screening enables pre-symptomatic treatment, improving outcomes and expanding market opportunity, particularly for infants and very young children.

5. Are there ongoing R&D efforts to improve upon nusinersen?
Yes, research into combination therapies, alternative delivery routes, and next-generation antisense molecules aims to enhance efficacy, reduce costs, and improve safety profiles.

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Sources

[1] Lefebvre, S., et al. (2017). “Spinal Muscular Atrophy.” Nature Reviews Disease Primers.
[2] Biogen. (2022). Annual Report 2021.
[3] Department of Health and Human Services. (2020). “Value-Based Pricing and Reimbursement Strategies for Rare Diseases.”