MIPOMERSEN SODIUM - Generic Drug Details

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

Mipomersen sodium is the generic ingredient in one branded drug marketed by Kastle Theraps Llc and is included in one NDA. There is one patent protecting this compound. Additional information is available in the individual branded drug profile pages.

Mipomersen sodium has forty-five patent family members in eleven countries.

Summary for MIPOMERSEN SODIUM

International Patents:	45
US Patents:	1
Tradenames:	1
Applicants:	1
NDAs:	1
Raw Ingredient (Bulk) Api Vendors:	2
Clinical Trials:	9
Patent Applications:	1,364
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for MIPOMERSEN SODIUM
What excipients (inactive ingredients) are in MIPOMERSEN SODIUM?	MIPOMERSEN SODIUM excipients list
DailyMed Link:	MIPOMERSEN SODIUM at DailyMed

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

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.

Recent Clinical Trials for MIPOMERSEN SODIUM

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

Sponsor	Phase
Kastle Therapeutics, LLC	Phase 1
Ionis Pharmaceuticals, Inc.	Phase 1
Ionis Pharmaceuticals, Inc.	Phase 3

See all MIPOMERSEN SODIUM clinical trials

Anatomical Therapeutic Chemical (ATC) Classes for MIPOMERSEN SODIUM

C10AX	Other lipid modifying agents
C10A	LIPID MODIFYING AGENTS, PLAIN
C10	LIPID MODIFYING AGENTS
C	Cardiovascular system

US Patents and Regulatory Information for MIPOMERSEN SODIUM

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Kastle Theraps Llc	KYNAMRO	mipomersen sodium	SOLUTION;SUBCUTANEOUS	203568-001	Jan 29, 2013		DISCN	Yes	No				Y

Expired US Patents for MIPOMERSEN SODIUM

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Kastle Theraps Llc	KYNAMRO	mipomersen sodium	SOLUTION;SUBCUTANEOUS	203568-001	Jan 29, 2013
Kastle Theraps Llc	KYNAMRO	mipomersen sodium	SOLUTION;SUBCUTANEOUS	203568-001	Jan 29, 2013
Kastle Theraps Llc	KYNAMRO	mipomersen sodium	SOLUTION;SUBCUTANEOUS	203568-001	Jan 29, 2013
Kastle Theraps Llc	KYNAMRO	mipomersen sodium	SOLUTION;SUBCUTANEOUS	203568-001	Jan 29, 2013
Kastle Theraps Llc	KYNAMRO	mipomersen sodium	SOLUTION;SUBCUTANEOUS	203568-001	Jan 29, 2013

EU/EMA Drug Approvals for MIPOMERSEN SODIUM

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Genzyme Europe BV	Kynamro	mipomersen sodium	EMEA/H/C/002429treatment of cholesterol and hypercholesterolaemia	Refused	no	no	no		2013-05-29

International Patents for MIPOMERSEN SODIUM

Country	Patent Number	Title	Estimated Expiration
World Intellectual Property Organization (WIPO)	03097662
South Africa	200503690	Antisense modulation of apolipoprotein B expression
Slovenia	2336318
Japan	4471650
Japan	2013066488	ANTISENSE MODULATION OF APOLIPOPROTEIN B EXPRESSION

Market Dynamics and Financial Trajectory for Mipomersen Sodium

Last updated: July 28, 2025

Introduction

Mipomersen sodium represents a targeted antisense oligonucleotide therapy designed to reduce low-density lipoprotein cholesterol (LDL-C) levels in patients with homozygous familial hypercholesterolemia (HoFH). Developed by Ionis Pharmaceuticals and marketed by Genzyme (a Sanofi company), mipomersen's journey through regulatory approval, commercialization, and market evolution provides critical insights into the complex landscape of specialty lipid-lowering therapies. This analysis explores the market forces, financial trends, regulatory influences, and competitive dynamics shaping mipomersen’s trajectory.

Market Overview and Therapeutic Context

Familial hypercholesterolemia (FH) affects approximately 1 in 250 individuals globally, with the homozygous form being particularly severe due to markedly elevated LDL-C levels and increased cardiovascular risk. Mipomersen emerged as a novel therapeutic option targeting the underlying genetic defect through antisense technology, aiming to provide a clinically significant reduction in LDL-C levels.

The therapy entered a niche, high-value segment, characterized by limited treatment options and unmet medical needs. Prior to mipomersen, lipoprotein apheresis and PCSK9 inhibitors served as the primary interventions for refractory cases, establishing a competitive yet distinct landscape.

Regulatory and Commercial Milestones

Regulatory Approval and Challenges

In 2013, the U.S. Food and Drug Administration (FDA) approved mipomersen for HoFH treatment, recognizing its potential in a highly challenging patient population. However, its approval carried significant restrictions, primarily due to safety concerns, notably hepatotoxicity risks. The European Medicines Agency (EMA) declined approval, citing safety and lack of sufficient efficacy data.

These regulatory hurdles significantly constrained mipomersen's market penetration. The limited approval scope and safety mandates resulted in restricted prescribing, impacting revenue viability.

Market Penetration and Adoption Dynamics

Despite regulatory endorsements, mipomersen's adoption remained modest. Physicians noted its efficacy in LDL-C reduction but expressed concern over adverse effects, particularly hepatotoxicity and injection site reactions. The requirement for regular injections (subcutaneous administration) further limited patient acceptance, especially when alternative therapies with better safety profiles emerged.

Pricing and Reimbursement Landscape

Pricing strategies for mipomersen targeted high-compliance, high-risk patients necessitating aggressive LDL-C lowering. However, reimbursement challenges arose due to safety concerns and competition from newer agents, complicating market access and profitability prospects.

Competitive Dynamics and Evolving Therapeutic Landscape

Emergence of PCSK9 Inhibitors

The advent of PCSK9 inhibitors (e.g., evolocumab, alirocumab) revolutionized hypercholesterolemia management. These monoclonal antibodies offer potent LDL-C reduction with favorable safety profiles and less invasive administration routes. Their rapid approval and adoption diminished mipomersen’s market share, especially considering the latter's safety and tolerability issues.

Gene Silencing and Future Therapies

Innovations in gene therapy and siRNA-based approaches continue to reshape the landscape. While mipomersen pioneered antisense technology for lipid disorders, emerging therapies with improved safety, efficacy, and convenience threaten its long-term relevance.

Financial Trajectory Analysis

Revenue Trends and Market Valuation

Mipomersen's initial launch projected substantial revenues given the limited options for HoFH. However, sales plateaued rapidly due to safety concerns, limited approval scope, and stiff competition. Internal estimates suggest peak annual sales remained below initial expectations, with some analysts estimating peak revenues in the range of $100-200 million globally (though actual realized revenue was significantly lower).

Research and Development and Lifecycle Considerations

Overall R&D investments for mipomersen's development and commercialization reflected a high-risk, high-cost profile. The limited market penetration diminished the return on investment, rendering mipomersen a niche asset with modest recurring revenue.

Shift in Market Dynamics

With the market’s evolution towards safer, more efficacious agents, mipomersen’s financial trajectory faced headwinds. Its role shifted to a niche therapy predominantly used under severe, refractory cases, constraining its market size and growth potential.

Regulatory Outlook and Future Prospects

Given the current market landscape, mipomersen’s future depends on several factors:

• Regulatory Decisions: Further restrictive indications or withdrawal risks persist, especially in regions with stringent safety standards.
• Market Receptivity: Prescriber and patient acceptance remains low, challenging expansion efforts.
• Reformulation and Combination Strategies: Potential development of next-generation antisense agents or combination therapies could revitalize its positioning, though such innovations require significant investment.

Conclusion: Market and Financial Overview

Mipomersen sodium exemplifies a high-risk, high-precision therapeutic whose initial promise met with regulatory and safety challenges, limiting widespread adoption. While it established proof-of-concept for antisense-based lipid modulation, its commercial and financial trajectory illustrates the importance of safety profile, patient convenience, and competitive innovation in determining long-term success. The drug’s niche role is unlikely to expand significantly without new formulations or indications; thus, its current and future value remains constrained within a specialized, low-growth segment.

Key Takeaways

• Regulatory restrictions and safety concerns curtailed mipomersen’s market potential, limiting revenue streams and adoption.
• Emergence of PCSK9 inhibitors significantly outpaced mipomersen in efficacy, safety, and convenience, relegating it to a niche status.
• Limited sales and reimbursement barriers underscored the challenges of high-cost, riskier biologic therapies in competitive markets.
• Future prospects hinge on innovation, potential new indications, or formulations that can mitigate safety issues and improve tolerability.
• The mipomersen case underscores the importance of balancing efficacy with safety and ease of use for successful market integration of high-value biologics.

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FAQs

1. Why did mipomersen face regulatory challenges despite FDA approval?
Because of safety concerns, particularly hepatotoxicity, which led to restrictions on its use and limited acceptance globally, notably with the EMA declining approval.

2. How does mipomersen compare to PCSK9 inhibitors in managing HoFH?
PCSK9 inhibitors offer greater efficacy, better safety profiles, and ease of administration, making them more favorable choices over mipomersen, which has safety and tolerability issues.

3. What has been the primary factor limiting mipomersen’s revenue growth?
Stringent safety concerns, limited approval scope, and competition from newer agents have constrained its market penetration and sales.

4. Are there prospects for mipomersen in future therapies?
Potential exists if new formulations reduce toxicity or if evidence emerges supporting wider indications, but current prospects are limited.

5. What lessons can pharmaceutical companies learn from mipomersen’s market trajectory?
Prioritizing safety and patient convenience, alongside efficacy, is crucial. Early and continuous safety evaluation can determine sustained market success for high-risk therapies.

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References

[1] FDA. (2013). FDA approves mipomersen, a new drug for homozygous familial hypercholesterolemia.
[2] EMA. (2014). Assessment report on mipomersen—European Medicines Agency.
[3] Ionis Pharmaceuticals. (2022). Mipomersen: Clinical data and market analysis.
[4] IQVIA. (2022). Global pharmaceutical market projections for lipid-lowering therapies.
[5] MarketWatch. (2023). Cytokine therapeutics and antisense drug market analysis.