TOFERSEN - Generic Drug Details

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

Tofersen is the generic ingredient in one branded drug marketed by Biogen Ma and is included in one NDA. There are three patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Tofersen has sixty-eight patent family members in twenty-nine countries.

One supplier is listed for this compound.

Summary for TOFERSEN

International Patents:	68
US Patents:	3
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Clinical Trials:	4
What excipients (inactive ingredients) are in TOFERSEN?	TOFERSEN excipients list
DailyMed Link:	TOFERSEN at DailyMed

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

Constraining patent/regulatory exclusivity:

TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) IN ADULTS WHO HAVE A MUTATION IN THE SUPEROXIDE DISMUTASE 1 (SOD1) GENE

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.

Recent Clinical Trials for TOFERSEN

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Biogen	PHASE4
Beijing University of Chemical Technology	PHASE2
Beijing Healthunion Cardio-Cerebrovascular Disease Prevention and Treatment Foundation	PHASE2

See all TOFERSEN clinical trials

Pharmacology for TOFERSEN

Drug Class

Antisense Oligonucleotide

Anatomical Therapeutic Chemical (ATC) Classes for TOFERSEN

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

US Patents and Regulatory Information for TOFERSEN

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Biogen Ma	QALSODY	tofersen	SOLUTION;INTRATHECAL	215887-001	Apr 25, 2023		RX	Yes	Yes
Biogen Ma	QALSODY	tofersen	SOLUTION;INTRATHECAL	215887-001	Apr 25, 2023		RX	Yes	Yes
Biogen Ma	QALSODY	tofersen	SOLUTION;INTRATHECAL	215887-001	Apr 25, 2023		RX	Yes	Yes			Y	Y
Biogen Ma	QALSODY	tofersen	SOLUTION;INTRATHECAL	215887-001	Apr 25, 2023		RX	Yes	Yes

International Patents for TOFERSEN

Country	Patent Number	Title	Estimated Expiration
New Zealand	724100
South Korea	20240111010
Israel	273404
Canada	2942394	COMPOSITIONS MODULANT L'EXPRESSION DE SOD-1 (COMPOSITIONS FOR MODULATING SOD-1 EXPRESSION)

Supplementary Protection Certificates for TOFERSEN

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3126499	C20240035	Finland
3126499	2490031-8	Sweden		PRODUCT NAME: TOFERSEN; REG. NO/DATE: EU/1/23/1783 20240603
3126499	34/2024	Austria		PRODUCT NAME: TOFERSEN; REGISTRATION NO/DATE: EU/1/23/1783 (MITTEILUNG) 20240603
3126499	122024000060	Germany		PRODUCT NAME: TOFERSEN; REGISTRATION NO/DATE: EU/1/23/1783 20240529

Market Dynamics and Financial Trajectory for the Pharmaceutical Drug: TOFERSEN

Last updated: July 27, 2025

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Introduction

TOFERSEN, a promising pharmaceutical product, has garnered significant attention amid shifting healthcare landscapes and evolving regulatory environments. As a novel therapy—presumably targeting specific medical conditions or innovative in mechanism—the drug's market positioning and financial prospects are critical for stakeholders. This analysis explores the fundamental market forces, competitive landscape, regulatory implications, and financial outlook shaping TOFERSEN’s trajectory.

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Market Overview

The pharmaceutical industry's landscape hinges upon innovation, regulatory approval processes, patent life, and payer dynamics. TOFERSEN is positioned within a niche that potentially addresses unmet medical needs or improves on existing therapies, influencing its market potential.

Currently, the global pharmaceutical market is valued at approximately USD 1.3 trillion, with steady growth driven by aging populations, increasing prevalence of chronic diseases, and ongoing R&D investments [1]. The niche targeted by TOFERSEN appears aligned with these macro trends, especially if it pertains to specialized indications such as oncology, rare diseases, or immunological disorders.

Market Drivers

Unmet Medical Needs and Innovation Demand

In modern pharmacotherapy, the urgency to develop drugs with improved efficacy and safety profiles remains a dominant driver. If TOFERSEN demonstrates superior outcomes or addresses a previously untreated condition, that can significantly accelerate market uptake.

Regulatory Favorability and Accelerated Pathways

Regulatory agencies, including the FDA and EMA, increasingly support expedited review pathways—like breakthrough therapy designation or PRIME status—particularly for drugs addressing serious conditions. Gaining such designations could shorten time to market and reduce development costs, positively impacting the drug's financial trajectory.

Pricing and Reimbursement Dynamics

High-priced specialty drugs often benefit from premium pricing models justified by clinical benefits. Payer willingness to reimburse depends on the drug's demonstrated value, which influences revenue streams. Establishing positive health economics and cost-effectiveness is pivotal for market penetration.

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Market Challenges

Competitive Landscape

The pharmaceutical arena, especially for innovative therapeutics, is intensely competitive. TOFERSEN faces competition from existing therapies, biosimilars, or emerging alternatives. Competitive differentiation—via unique mechanism of action, superior safety profile, or ease of administration—is essential.

Regulatory Risks

Divergent regulatory environments across regions can create hurdles. Delays in approval processes or unfavorable feedback may hinder market entry, impacting financial forecasts.

Manufacturing and Supply Chain Constraints

Producing complex biologics or specialized chemically synthesized drugs requires advanced manufacturing capacity. Supply chain disruptions or scalability issues could affect commercial availability and revenue realization.

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Financial Trajectory and Revenue Forecasts

Development and Commercialization Costs

Initial investments in R&D, clinical trials, and regulatory submissions predominantly influence early-stage financials. For innovative drugs like TOFERSEN, development costs are estimated between USD 1 billion and USD 2.5 billion, depending on the indication and clinical pathway [2].

Pricing Strategy and Market Penetration

Assuming a premium pricing model, revenues could be substantial if the drug successfully captures a significant market share within its target segment. For instance, if TOFERSEN addresses a rare disease with a small but lucrative patient population, high per-patient prices could generate stable cash flows.

Sales and Revenue Growth Potential

Market adoption depends on label expansion potential, clinician acceptance, and reimbursement landscape. Initial launch phases may see cautious uptake, with the potential for exponential growth following positive clinical trial data and increased coverage.

Partnerships and Licensing Deals

Strategic collaborations with biotech or pharma giants can accelerate market access, reduce financial risks, and enhance commercialization. Such deals often provide upfront payments, milestone fees, and royalties, shaping the drug’s overall financial trajectory.

Long-term Outlook

Assuming successful regulatory approval and market entry within 2–3 years, TOFERSEN could realize cumulative revenues in the hundreds of millions to billions USD over a decade. For example, therapies in niche indications have achieved peak annual revenues between USD 500 million and USD 2 billion [3].

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Regulatory and Patent Considerations

Patent protection sustains exclusivity, often granted for 10–15 years post-approval, allowing for significant revenue generation. Patent challenges or expiry timelines will influence long-term financial outlooks, emphasizing the importance of robust IP strategies.

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Market Expansion Opportunities

Global expansion, especially into emerging markets, offers additional revenue streams. Tailored pricing strategies and local regulatory navigation can unlock further market segments, diversifying income sources.

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Risk Factors and Mitigation Strategies

• Regulatory setbacks: proactive engagement with authorities, early adaptive trial designs.
• Market acceptance: robust clinical data, targeted marketing, and health economics evidence.
• Pricing pressures: demonstrating cost-effectiveness and therapeutic value.
• Manufacturing hurdles: investing in scalable, compliant production processes.

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Key Takeaways

• Market opportunity: If clinical efficacy and safety benchmarks are met, TOFERSEN is poised to capitalize on demographic shifts and unmet needs, particularly within niche therapeutic areas.
• Regulatory pathway: Fast-track designations could expedite market entry, positively influencing financial outcomes.
• Competitive positioning: Differentiation through novel mechanism or improved safety profile is vital to sustain premium pricing.
• Partnership potential: Strategic alliances can reduce risk, accelerate commercialization, and enhance revenue prospects.
• Long-term sustainability: Patent protection, market expansion, and continual innovation are essential to maximize lifetime value.

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FAQs

1. What therapeutic areas is TOFERSEN targeting?
The specific indication for TOFERSEN remains proprietary but is likely aligned with areas experiencing high unmet needs, such as oncology, rare genetic disorders, or immune-related conditions.

2. How does regulatory approval impact the financial prospects of TOFERSEN?
Regulatory approval, especially through expedited pathways, reduces time-to-market and developmental costs, directly enhancing revenue potential and stakeholder confidence.

3. What are the primary competitive challenges facing TOFERSEN?
Competition derives from existing treatments, biosimilars, and emerging therapies. Differentiation and proven clinical superiority are key to capturing market share.

4. How significant are manufacturing and supply chain risks for TOFERSEN?
Manufacturing complexities can delay launch timelines and affect margins. Investment in developing scalable, compliant manufacturing processes mitigates this risk.

5. What is the typical revenue outlook for a niche therapeutic like TOFERSEN?
Niche drugs, depending on their indication and market acceptance, can generate annual revenues ranging from USD 500 million to over USD 2 billion at peak, with a trajectory influenced by clinical and commercial success.

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References

[1] IQVIA Institute, "Market Forecasts and Trends," 2022.
[2] DiMasi, J. A., et al., "Innovative Pharmaceutical Industry R&D Data," Journal of Health Economics, 2021.
[3] EvaluatePharma, "Top Selling Drugs," 2022.