Tofersen - Generic Drug Details

What are the generic drug 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 patent family members in twenty-six countries.

One supplier is listed for this compound.

Summary for tofersen

International Patents:	60
US Patents:	3
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Clinical Trials:	2
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
Ionis Pharmaceuticals, Inc.	Phase 3
Biogen	Phase 3

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	10,669,546
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	10,385,341		Y	Y

International Patents for tofersen

Country	Patent Number	Title	Estimated Expiration
China	106459972	用于调节SOD‑1表达的组合物 (Compositions for modulating SOD-1 expression)
Mexico	2020007166	COMPOSICIONES PARA MODULAR LA EXPRESION DE SOD-1. (COMPOSITIONS FOR MODULATING SOD-1 EXPRESSION.)
Australia	2019268063

Supplementary Protection Certificates for tofersen

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3126499	301293	Netherlands		PRODUCT NAME: TOFERSEN; REGISTRATION NO/DATE: EU/1/23/1783 20240603
3126499	CR 2024 00038	Denmark		PRODUCT NAME: TOFERSEN; REG. NO/DATE: EU/1/23/1783 20240603

Tofersen Market Analysis and Financial Projection

Market Dynamics and Financial Trajectory for Tofersen

Introduction to Tofersen

Tofersen, developed by Biogen, is an investigational antisense drug designed to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), a rare and devastating form of the disease. Here, we delve into the market dynamics and financial trajectory of this groundbreaking medication.

Mechanism of Action and Clinical Trials

Tofersen works by targeting the SOD1 gene mutation, which is responsible for a small fraction of ALS cases. The Phase 3 VALOR study, although not meeting its primary endpoint, showed signs of slowing disease progression across multiple secondary and exploratory endpoints[2].

Regulatory Milestones

Despite the mixed results from the Phase 3 trial, Biogen pursued an accelerated approval pathway based on biomarker data, specifically the reduction in neurofilament light chain levels, which is a marker of disease progression. This approach was successful, and the FDA approved tofersen in a precedent-setting decision, marking the first ALS drug approved based on biomarker data[3].

Market Size and Forecast

The market for tofersen is expected to grow, albeit within a niche segment due to its specificity for SOD1-ALS patients. The report from ResearchAndMarkets provides a detailed market forecast for tofersen across the 7MM (United States, EU5, and Japan) from 2019 to 2032. Here are some key market size projections:

• United States: The market size for tofersen in the U.S. is anticipated to increase significantly over the forecast period, driven by the high unmet medical need and incremental healthcare spending[1].
• EU5: Countries such as Germany, France, Italy, Spain, and the United Kingdom will also see growth, with detailed market size projections available for each country[1].
• Japan: The Japanese market is expected to contribute to the overall growth, reflecting the global trend of increasing investment in ALS treatments[1].

Competitive Landscape

Tofersen faces competition from other emerging therapies for ALS, including Relyvrio and Radicava. However, its unique mechanism of action targeting SOD1 mutations sets it apart. The competitive landscape includes both marketed therapies and emerging products, which will continue to shape the market dynamics[1].

Pricing Strategy

Biogen has announced that the pricing for tofersen will be within a range comparable to other recently launched ALS treatments. For example, Relyvrio costs about $158,000 per year, and an oral version of Radicava costs about $170,000 annually. This pricing strategy aims to balance innovation with patient access[3].

Financial Projections

Analysts predict that tofersen will generate around $150 million in annual sales at its peak, given its limited target population. This figure, while modest compared to broader market drugs, reflects the significant unmet need and the willingness of patients and healthcare systems to invest in effective treatments for rare diseases[3].

Real-World Evidence and Clinical Outcomes

A recent cohort study from the German early access program provided real-world evidence of tofersen's effectiveness. The study showed a decrease in neurofilament light chain (NfL) serum levels and a reduction in phosphorylated neurofilament heavy chain (pNfH) in cerebrospinal fluid (CSF), indicating a slowing of disease progression. The median progression rate under tofersen treatment was 0.11 points of ALSFRS-R lost per month, further supporting its clinical benefit[4].

Expansion of Early Access Programs

Given the positive outcomes and the high unmet medical need, Biogen has expanded its early access program (EAP) to include the broader SOD1-ALS population. This move is expected to increase patient access and further validate the drug's efficacy in real-world settings[2].

Future Market Assessments

The market for ALS treatments is expected to evolve significantly with the introduction of new therapies and the advancement of existing ones. Tofersen's approval sets a precedent for the use of biomarker data in drug approvals, which could expedite the development and approval of future ALS treatments. The ongoing research and development activities, including Biogen's broad pipeline of investigational drugs, will continue to shape the market dynamics[1][3].

Key Takeaways

• Regulatory Approval: Tofersen is the first ALS drug approved based on biomarker data, setting a new pathway for future approvals.
• Market Size: Expected to grow significantly across the 7MM, with peak annual sales projected at around $150 million.
• Pricing: Comparable to other ALS treatments, balancing innovation with patient access.
• Clinical Outcomes: Real-world evidence shows a slowing of disease progression and reduction in biomarkers.
• Competitive Landscape: Unique mechanism targeting SOD1 mutations, with competition from other emerging therapies.
• Future Market: Expected evolution with new therapies and advancements in existing treatments.

FAQs

1. What is Tofersen and how does it work? Tofersen is an antisense drug developed by Biogen to treat SOD1-ALS by targeting the SOD1 gene mutation, thereby reducing the production of the toxic SOD1 protein.

2. What were the results of the Phase 3 VALOR study? The Phase 3 VALOR study did not meet its primary endpoint, but it showed signs of slowing disease progression across multiple secondary and exploratory endpoints.

3. How was Tofersen approved by the FDA? Tofersen was approved through an accelerated approval pathway based on biomarker data, specifically the reduction in neurofilament light chain levels.

4. What is the pricing strategy for Tofersen? The pricing for Tofersen is within a range comparable to other recently launched ALS treatments, such as Relyvrio and Radicava.

5. What are the financial projections for Tofersen? Analysts predict that Tofersen will generate around $150 million in annual sales at its peak, given its limited target population.

Sources

1. ResearchAndMarkets: "Tofersen Emerging Drug Insight and Market Forecast - 2032"
2. Biogen: "Biogen Announces Topline Results from the Tofersen Phase 3 Study and its Open-Label Extension in SOD1-ALS"
3. Biopharma Dive: "FDA approves new ALS medicine in precedent-setting decision"
4. The Lancet: "Effects of tofersen treatment in patients with SOD1-ALS in a “real-world” setting – a 12-month multicenter cohort study from the German early access program"