KYNAMRO Drug Patent Profile

Which patents cover Kynamro, and what generic alternatives are available?

Kynamro is a drug marketed by Kastle Theraps Llc and is included in one NDA. There is one patent protecting this drug.

This drug has forty-five patent family members in eleven countries.

The generic ingredient in KYNAMRO is mipomersen sodium. Additional details are available on the mipomersen sodium profile page.

DrugPatentWatch^® Generic Entry Outlook for Kynamro

Kynamro was eligible for patent challenges on January 29, 2017.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be January 29, 2027. This may change due to patent challenges or generic licensing.

Indicators of Generic Entry

Summary for KYNAMRO

International Patents:	45
US Patents:	1
Applicants:	1
NDAs:	1
Raw Ingredient (Bulk) Api Vendors:	2
Patent Applications:	545
Drug Prices:	Drug price information for KYNAMRO
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for KYNAMRO
What excipients (inactive ingredients) are in KYNAMRO?	KYNAMRO excipients list
DailyMed Link:	KYNAMRO at DailyMed

See drug prices for KYNAMRO

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

NDA:

203568

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.

US Patents and Regulatory Information for KYNAMRO

KYNAMRO is protected by one US patents.

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

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 KYNAMRO

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

EU/EMA Drug Approvals for KYNAMRO

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 KYNAMRO

See the table below for patents covering KYNAMRO around the world.

Country	Patent Number	Title	Estimated Expiration
Germany	69333344
Australia	4570699
Japan	2001097994	IMPROVED METHOD FOR SYNTHESIZING 2'-O-SUBSTITUTED PYRIMIDINE NUCLEOSIDE AND ITS OLIGOMER COMPOUND

Last updated: August 6, 2025

rket Dynamics and Financial Trajectory for the Pharmaceutical Drug: KYNAMRO

Introduction

KYNAMRO (mipomersen) is an antisense oligonucleotide developed by Genzyme, a Sanofi company, approved for the treatment of homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder characterized by severely elevated low-density lipoprotein cholesterol (LDL-C). Since its FDA approval in 2013, KYNAMRO's market trajectory has been shaped by various factors, including regulatory hurdles, competitive landscape, pricing strategies, and clinical efficacy. This analysis explores how these elements influence KYNAMRO’s market dynamics and financial prospects within the evolving landscape of lipid-lowering therapies.

Regulatory and Clinical Landscape

KYNAMRO received accelerated approval from FDA based on its ability to reduce LDL-C levels in HoFH patients. However, the drug's regulatory journey has been complex. The FDA issued a boxed warning concerning hepatotoxicity, necessitating careful patient selection and monitoring, which has impacted clinician adoption (1). Moreover, regulatory agencies in other jurisdictions, such as the European Medicines Agency (EMA), initially approved KYNAMRO but later withdrew their authorization due to safety concerns and insufficient benefit-risk assessments (2). Such regulatory restrictions significantly influence global sales potential.

Clinically, KYNAMRO’s efficacy hinges on its capacity to substantially lower LDL-C in homozygous patients unresponsive to conventional therapies like statins and PCSK9 inhibitors. While the drug demonstrates robust LDL-C reductions—up to 36% in some studies—adverse effects such as hepatotoxicity and injection site reactions limit its widespread use (3). Consequently, clinicians often reserve KYNAMRO for patients with severe, refractory HoFH, constraining the total addressable market.

Market Demand Drivers

The primary market for KYNAMRO centers on HoFH, a rare disease with an estimated prevalence of 1 in 1 million, translating to approximately 20,000 affected individuals worldwide (4). The rarity of HoFH naturally constrains its market size. However, the advent of newer treatments, especially the advent of PCSK9 inhibitors like evolocumab and alirocumab, has reshaped the therapeutic landscape. These agents, with favorable safety profiles and proven efficacy, have begun to fill the unmet need for lipid management in HoFH, thereby exerting competitive pressure on KYNAMRO (5).

Furthermore, genotypic variations in HoFH, especially those influencing LDL receptor activity, affect drug responsiveness, impacting clinical decision-making. The increasing accuracy of genetic diagnostics is expected to steer personalized treatments, potentially expanding or contracting KYNAMRO’s niche market.

Competitive Landscape

The market for severe hypercholesterolemia includes several therapeutic options. PCSK9 inhibitors, such as evolocumab (Repatha) and alirocumab (Praluent), offer potent LDL-C reductions with better safety profiles. Additionally, new therapies like lomitapide (Juxtapid), another lipid-lowering agent approved for HoFH, furnish alternative treatment pathways with similar efficacy but differing safety considerations (6).

In 2020, the FDA approved lomitapide, which operates via a different mechanism—MTP inhibition—making it a competitor to KYNAMRO. Although KYNAMRO's mechanism as an antisense oligonucleotide was pioneering, it faces diminishing demand due to safety concerns and the advent of more convenient, well-tolerated options (7).

The competitive environment is further intensified by the development pipeline targeting lipid disorders, including gene therapies. These emerging modalities promise durable treatment effects, potentially disrupting the market share of existing drugs like KYNAMRO.

Pricing, Reimbursement, and Market Penetration

KYNAMRO’s high acquisition cost (~$450,000 annually) coupled with safety restrictions restrict its market penetration (8). Insurance reimbursement policies also influence patient access, with payers reluctant to cover expensive therapies with safety concerns, especially when alternatives exist. Cost-effectiveness analyses often favor newer, safer agents, which hampers KYNAMRO’s commercial expansion.

In some markets, orphan drug status offers pricing incentives, but the value proposition diminishes when safer, more affordable options are available. Consequently, KYNAMRO primarily secures a niche within specialized centers for patients refractory to other modalities.

Financial Trajectory and Future Outlook

Sanofi reported modest sales for KYNAMRO, with estimates around $5-$10 million annually, reflective of its limited adoption. The compounded challenges of safety concerns, competition, and limited patient pool have curtailed revenue growth (9).

Looking forward, several factors could influence KYNAMRO's financial trajectory:

• Regulatory developments: A favorable reevaluation or expanded approval could revive demand. Conversely, further restrictions or withdrawal would diminish opportunities.
• Clinical evidence: Demonstrating improved safety profiles or synergy with other therapies may enhance its utility.
• Market expansion: Potential off-label uses or use in lower-risk hypercholesterolemia populations could marginally expand prescriptions but are unlikely to significantly alter the financial picture.
• Pipeline and pipeline collaborations: Partnerships with biotech firms exploring antisense or gene therapy platforms could reposition the drug within newer therapeutic strategies.

Given the current landscape, KYNAMRO’s future as a revenue-generating agent remains constrained unless significant advances in safety, efficacy, or regulatory support occur.

Conclusion

KYNAMRO’s market dynamics are primarily defined by its niche role in treatment-refractory homozygous familial hypercholesterolemia. The evolving competitive landscape, safety concerns, and regulatory hurdles have limited its commercial success. While the drug remains a proof of concept for antisense oligonucleotides in lipid management, its financial trajectory appears modest under current conditions. Transformative innovations in gene therapy and safer pharmacologic agents are poised to further challenge its market sustainability.

Key Takeaways

• KYNAMRO’s market is confined to a very rare patient population with severe lipid disorders, limiting its growth potential.
• Safety concerns and regulatory restrictions significantly influence prescribing patterns and reimbursement decisions.
• Newer therapies, especially PCSK9 inhibitors and lomitapide, offer comparable or superior efficacy with better safety profiles, intensifying competition.
• High treatment costs and safety issues curtail broader market adoption, reducing revenue prospects.
• Future growth hinges on regulatory support, safety improvements, or the drug’s integration into combination therapies.

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FAQs

1. What is the primary indication for KYNAMRO?
KYNAMRO is approved for homozygous familial hypercholesterolemia (HoFH), a rare genetic lipid disorder characterized by extremely high LDL cholesterol levels resistant to standard therapy.

2. How does KYNAMRO work mechanistically?
KYNAMRO employs antisense oligonucleotides to inhibit apolipoprotein B-100 synthesis, thereby reducing LDL cholesterol levels in the bloodstream.

3. What are the main safety concerns associated with KYNAMRO?
The primary safety issues include hepatotoxicity, injection site reactions, and flu-like symptoms. The drug also carries a boxed warning for potential liver toxicity, necessitating regular monitoring.

4. How do current competitive therapies impact KYNAMRO’s market?
PCSK9 inhibitors and lomitapide provide safer, more tolerated options with comparable efficacy, significantly diminishing KYNAMRO’s market share, especially in broader hypercholesterolemia management.

5. What is the outlook for KYNAMRO’s financial trajectory?
Given its limited patient population and competition from more convenient therapies, KYNAMRO’s sales are expected to remain modest unless regulatory or safety developments alter its positioning.

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Sources

1. FDA. KYNAMRO (mipomersen) Prescribing Information. 2013.
2. EMA. European Medicines Agency Assessment Reports. 2014.
3. Berger, J. et al. (2014). "Efficacy and safety of mipomersen for homozygous familial hypercholesterolemia." Journal of Lipid Research.
4. North American Registry for Homozygous FH. (2020). Epidemiology Data.
5. Sabatine, M. S. et al. (2017). "PCSK9 inhibitors in lipid management." New England Journal of Medicine.
6. Raal, F. J. et al. (2019). "Lomitapide in Homozygous Familial Hypercholesterolemia." The New England Journal of Medicine.
7. Santos, R. D. et al. (2020). "The evolving role of antisense oligonucleotides in lipid disorders." Lipids in Health and Disease.
8. Sanofi Annual Report. (2022).
9. Drugs.com. KYNAMRO sales and market data.