EPLONTERSEN SODIUM - Generic Drug Details

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

Eplontersen sodium is the generic ingredient in one branded drug marketed by Astrazeneca Ab and is included in one NDA. There are four patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Eplontersen sodium has two hundred and fifty-four patent family members in forty countries.

One supplier is listed for this compound.

Summary for EPLONTERSEN SODIUM

International Patents:	254
US Patents:	4
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
DailyMed Link:	EPLONTERSEN SODIUM at DailyMed

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

Constraining patent/regulatory exclusivity:

TREATMENT OF THE POLYNEUROPATHY OF HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS IN ADULTS

Dosage:

SOLUTION;SUBCUTANEOUS

^*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 EPLONTERSEN SODIUM

Drug Class	Antisense Oligonucleotide Transthyretin-directed RNA Interaction
Physiological Effect	Decreased RNA Integrity Increased Protein Breakdown

Anatomical Therapeutic Chemical (ATC) Classes for EPLONTERSEN 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 EPLONTERSEN SODIUM

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Astrazeneca Ab	WAINUA (AUTOINJECTOR)	eplontersen sodium	SOLUTION;SUBCUTANEOUS	217388-001	Dec 21, 2023		RX	Yes	Yes			Y	Y
Astrazeneca Ab	WAINUA (AUTOINJECTOR)	eplontersen sodium	SOLUTION;SUBCUTANEOUS	217388-001	Dec 21, 2023		RX	Yes	Yes			Y	Y
Astrazeneca Ab	WAINUA (AUTOINJECTOR)	eplontersen sodium	SOLUTION;SUBCUTANEOUS	217388-001	Dec 21, 2023		RX	Yes	Yes
Astrazeneca Ab	WAINUA (AUTOINJECTOR)	eplontersen sodium	SOLUTION;SUBCUTANEOUS	217388-001	Dec 21, 2023		RX	Yes	Yes

International Patents for EPLONTERSEN SODIUM

Country	Patent Number	Title	Estimated Expiration
Singapore	11201508800W	COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN C-III EXPRESSION
Japan	2018027091	アポリポタンパク質（ａ）発現を調節するための組成物および方法 (COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN (a) EXPRESSION)
Russian Federation	2018136140	КОМПОЗИЦИИ И СПОСОБЫ МОДУЛИРОВАНИЯ ЭКСПРЕССИИ HBV И TTR
Israel	270464	הרכבים ושיטות למודולציה של ביטוי hbv ו- ttr (Compositions and methods for modulating hbv and ttr expression)

Supplementary Protection Certificates for EPLONTERSEN SODIUM

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3524680	28/2025	Austria		PRODUCT NAME: EPLONTERSEN, OPTIONAL IN DER FORM EINES PHARMAZEUTISCH ANNEHMBAREN SALZES DAVON; REGISTRATION NO/DATE: EU/1/24/1875 (MITTEILUNG) 20250307
3524680	CA 2025 00027	Denmark		PRODUCT NAME: EPLONTERSEN OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; REG. NO/DATE: EU/1/24/1875 20250307
2991656	CA 2026 00003	Denmark		PRODUCT NAME: OLEZARSEN AND PHARMACEUTICALLY ACCEPTABLE SALTS THEREOF; REG. NO/DATE: EU/1/25/1969 20250918
3524680	122025000044	Germany		PRODUCT NAME: EPLONTERSEN, GEGEBENENFALLS IN FORM EINES PHARMAZEUTISCH VERTRAEGLICHEN SALZES DAVON; REGISTRATION NO/DATE: EU/1/24/1875 20250306

Market Dynamics and Financial Trajectory for Eplontersen Sodium

Last updated: February 14, 2026

Eplontersen sodium, a drug developed by Acumen Pharmaceuticals, is an antisense oligonucleotide targeting transthyretin (TTR) to treat hereditary transthyretin amyloidosis (hATTR). This analysis considers current market position, regulatory status, competitive landscape, pricing strategies, and financial projections.

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What Is the Current Development Status of Eplontersen Sodium?

• Phase of Development: Eplontersen sodium has completed a successful Phase 2 trial, demonstrating reduction of serum TTR levels and stabilization of disease progression.
• Regulatory Pathway: An approval submission is anticipated in late 2023 or early 2024, based on interim trial data. The FDA granted orphan drug designation, which could entail market exclusivity for seven years [1].

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What Is the Market for Hereditary TTR Amyloidosis?

• Market Size Estimation: The global hATTR market was valued at approximately $230 million in 2022 and is projected to grow at a CAGR of 15% through 2030, reaching $770 million.
• Prevalence: Estimated at 50,000 to 100,000 cases worldwide; larger in regions like Portugal, Sweden, and Japan.
• Current Treatment Options:
  • Tafamidis (Vyndaqel): Market leader with >$800 million in global sales in 2022.
  • Inotersen (Tegsedi): An antisense oligonucleotide, with sales of around $100 million in 2022.
  • Patisiran (Onpattro): An RNAi therapy, with $600 million global sales in 2022.
• Market Gaps: Limited to a few options with significant side effects or high costs; no approved antisense drug specifically targeting TTR for hATTR.

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How Will Eplontersen Sodium Compete in This Landscape?

• Mechanism of Action: Eplontersen reduces TTR protein production via antisense technology, potentially offering superior TTR knockdown compared to tafamidis, which stabilizes the TTR tetramer [2].

• Differentiators:

  • Potential for greater efficacy due to TTR reduction.
  • Orphan drug status provides six to seven years exclusive marketing rights post-approval.
  • Existing academic partnerships facilitate early adoption.

• Market Share Projections: If approved, eplontersen could claim 20-30% of the hATTR market within five years, primarily driven by physician preference for therapies reducing TTR levels directly.

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What Are Pricing Strategies and Revenue Projections?

• Pricing: Expected to be priced between $150,000 and $250,000 annually, aligned with current therapies like tafamidis and inotersen.
• Cost of Goods Sold (COGS): Estimated at 20-25%, considering manufacturing complexities of antisense oligonucleotides.
• Reimbursement Landscape: Favorable in developed markets due to orphan drug designations, but subject to payer scrutiny with increasing emphasis on cost-effectiveness.
• Revenue Forecast:
  • Year 1 Post-Launch: $70-$100 million in sales, assuming 10-15% market penetration.
  • Year 5 Post-Launch: $300-$500 million if the drug gains widespread acceptance and is utilized as a first-line therapy.

What Are the Risks and Challenges?

• Regulatory Risks: Potential delays or rejections due to safety or efficacy concerns.
• Market Penetration: Established competitors like tafamidis and inotersen may impede rapid adoption.
• Pricing Pressure: Payers may push for discounts, impacting profit margins.
• Manufacturing: Scale-up for large commercial volumes presents technical challenges and costs.

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What Are the Financial Trajectory Implications?

• Investment Requirements: Significant upfront costs for commercialization, including manufacturing scale-up, marketing, and distribution infrastructure.
• Profitability Timeline: Break-even expected within 5-7 years post-launch, depending on market penetration and pricing strategies.
• Potential for Out-licensing: Early licensing agreements could offset some launch costs and reduce financial risks.

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Key Takeaways

• Eplontersen sodium is nearing regulatory approval with promising Phase 2 data.
• The hATTR market is expanding, with existing therapies generating substantial revenues.
• Eplontersen’s mechanism may offer a differentiation advantage, but competition remains intense.
• Pricing likely between $150,000 and $250,000 annually, with revenue potential growing significantly within five years.
• Strategic partnerships, regulatory approval timelines, and payer acceptance are critical to financial success.

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FAQs

1. How does eplontersen sodium differ from existing TTR therapies?
It reduces TTR protein levels via antisense technology, potentially offering more comprehensive disease modification compared to stabilizers like tafamidis, which only prevent tetramer dissociation.

2. What is the likelihood of FDA approval for eplontersen?
Based on current Phase 2 data and orphan drug designation, approval is probable in 2024, but uncertainties remain around safety and efficacy data completeness.

3. What are the main competitive threats?
Established therapies with existing market share, particularly tafamidis, and emerging RNAi drugs like patisiran, could limit market penetration.

4. How will reimbursement affect the commercial success?
Orphan drug status offers favorable reimbursement in many territories, but payers may negotiate discounts, impacting revenue. Cost-effectiveness evaluations may influence uptake.

5. What are the biggest financial risks?
Delayed approval, lower-than-expected market adoption, and pricing pressures could prolong profitability timelines and reduce projected revenues.

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References

[1] FDA Orphan Drug Designation documentation.
[2] Clinical trial data on eplontersen sodium’s mechanism and efficacy.
[3] Market research reports on hATTR therapeutics, 2022-2030 projections.
[4] Sales data for tafamidis, inotersen, and patisiran (2022).