Mipomersen sodium - Generic Drug Details

What are the generic 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:	1
Clinical Trials:	9
Patent Applications:	857
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
Ionis Pharmaceuticals, Inc.	Phase 1
Kastle Therapeutics, LLC	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/002429 treatment of cholesterol and hypercholesterolaemia	Refused	no	no	no		2013-05-29

International Patents for mipomersen sodium

Country	Patent Number	Title	Estimated Expiration
Canada	2455228	MODULATION ANTISENS DE L'EXPRESSION DE L'APOLIPOPROTEINE B (ANTISENSE MODULATION OF APOLIPOPROTEIN B EXPRESSION)
Denmark	2336318
European Patent Office	2174945	Modulation par anti-sense de l'expression de apolipoprotéine B (Antisense modulation of apolipoprotein B expression)
Japan	2013066488	ANTISENSE MODULATION OF APOLIPOPROTEIN B EXPRESSION
European Patent Office	1419168	MODULATION ANTISENS DE L'EXPRESSION DE L'APOLIPOPROTEINE B (ANTISENSE MODULATION OF APOLIPOPROTEIN B EXPRESSION)

Mipomersen sodium Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Mipomersen Sodium

Introduction

Mipomersen sodium, marketed under the brand name Kynamro, is a synthetic phosphorothioate oligonucleotide used for the treatment of homozygous familial hypercholesterolemia (HoFH). Here, we delve into the market dynamics and financial trajectory of this drug, highlighting its development, regulatory approvals, market performance, and the challenges it has faced.

Development and Regulatory Approval

Mipomersen sodium was developed by Ionis Pharmaceuticals (formerly known as Isis Pharmaceuticals) and later licensed to Genzyme Corporation. The drug underwent extensive clinical trials, including Phase 3 studies, which demonstrated its efficacy in reducing low-density lipoprotein cholesterol (LDL-C) and apolipoprotein B (apo B) levels[4][5].

In 2013, the FDA approved mipomersen sodium as an adjunct therapy to maximally tolerated lipid-lowering medications and diet for patients with HoFH. However, the approval came with a black box warning due to the risk of liver toxicity, including elevations in liver enzymes and hepatic steatosis[2][4].

Market Dynamics

Target Market

Mipomersen sodium is specifically indicated for patients with HoFH, a rare genetic disorder characterized by extremely high levels of LDL-C. This niche market, although small, presents a significant unmet medical need, making mipomersen a valuable therapeutic option for these patients[2][5].

Competition

The market for lipid-lowering therapies is highly competitive, with established drugs such as statins and newer agents like PCSK9 inhibitors. However, mipomersen's unique mechanism of action, which does not depend on LDL receptor function, positions it as a distinct option for patients who do not respond adequately to other treatments[4].

Pricing and Access

Given the rare nature of HoFH and the significant clinical benefit mipomersen offers, the drug is priced accordingly. However, the high cost and the need for it to be administered under the Kynamro Risk Evaluation and Mitigation Strategy (REMS) program due to its hepatotoxicity risk can limit its accessibility[2].

Financial Trajectory

Licensing and Partnerships

Ionis Pharmaceuticals entered into a strategic alliance with Genzyme Corporation, which included a $150 million equity investment, an upfront licensing fee of $175 million, and potential milestone payments of over $1.5 billion. This partnership was crucial for the commercialization of mipomersen and provided significant financial backing to Ionis[3].

Clinical Success and Milestone Payments

The clinical success of mipomersen in Phase 3 trials added significant value to Ionis's drug development pipeline. The company received milestone payments as part of its partnership with Genzyme, contributing to its financial growth. For instance, Ionis received approximately $270 million from these partnerships by early 2008[3].

Revenue and Profit Sharing

Ionis was set to share in the future commercial success of mipomersen through profit sharing or royalties. However, the drug's market performance was impacted by its safety concerns and the restricted access program, which may have limited its revenue potential[3].

Challenges and Market Withdrawal

Hepatotoxicity Risk

The most significant challenge faced by mipomersen sodium was its association with liver toxicity. Despite its efficacy, the risk of hepatotoxicity led to a black box warning and the implementation of a REMS program. This significantly limited its market adoption and ultimately led to its withdrawal from the market due to these safety concerns[2][4].

Market Impact

The withdrawal of mipomersen sodium from the market had a negative impact on Ionis's financial trajectory. The loss of a key drug from their portfolio reduced potential revenue streams and affected investor confidence. However, Ionis has continued to develop other drugs and maintain a robust pipeline, mitigating some of the financial impact[3].

Key Takeaways

• Development and Approval: Mipomersen sodium was approved by the FDA in 2013 for the treatment of HoFH but came with a black box warning for liver toxicity.
• Market Dynamics: The drug targeted a niche market with significant unmet medical need but faced competition from other lipid-lowering therapies.
• Financial Trajectory: The drug generated significant financial milestones through licensing and partnerships but was ultimately withdrawn from the market due to safety concerns.
• Challenges: Hepatotoxicity risk was a major challenge that limited market adoption and led to the drug's withdrawal.

FAQs

What is mipomersen sodium used for?

Mipomersen sodium is used as an adjunct therapy to maximally tolerated lipid-lowering medications and diet for the treatment of homozygous familial hypercholesterolemia (HoFH).

Why was mipomersen sodium withdrawn from the market?

Mipomersen sodium was withdrawn from the market due to its association with liver toxicity, including elevations in liver enzymes and hepatic steatosis.

What is the mechanism of action of mipomersen sodium?

Mipomersen sodium works by inhibiting the synthesis of apolipoprotein B-100 (apo B), leading to a decrease in LDL-C and other apo B-containing lipoproteins.

Who developed and marketed mipomersen sodium?

Mipomersen sodium was developed by Ionis Pharmaceuticals and marketed by Genzyme Corporation under the brand name Kynamro.

What were the financial implications of mipomersen sodium's market performance?

The drug generated significant financial milestones through licensing and partnerships but its market withdrawal due to safety concerns negatively impacted Ionis's financial trajectory.

Sources

1. Synapse: Mipomersen sodium - Drug Targets, Indications, Patents.
2. DrugBank: Mipomersen: Uses, Interactions, Mechanism of Action.
3. Ionis Pharmaceuticals: United States Securities and Exchange Commission Form 10-K.
4. FDA: 203568Orig1s000 - Clinical Pharmacology Review.
5. WVU Pharmacy: Mipomersen Sodium Manufacturer: Genzyme Corp. Drug Class.