Lomitapide mesylate - Generic Drug Details

What are the generic sources for lomitapide mesylate and what is the scope of patent protection?

Lomitapide mesylate is the generic ingredient in one branded drug marketed by Chiesi and is included in one NDA. There are eight patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Lomitapide mesylate has twenty-seven patent family members in eighteen countries.

There are two drug master file entries for lomitapide mesylate. Three suppliers are listed for this compound.

Summary for lomitapide mesylate

International Patents:	27
US Patents:	8
Tradenames:	1
Applicants:	1
NDAs:	1
Drug Master File Entries:	2
Finished Product Suppliers / Packagers:	3
Raw Ingredient (Bulk) Api Vendors:	54
Patent Applications:	385
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for lomitapide mesylate
What excipients (inactive ingredients) are in lomitapide mesylate?	lomitapide mesylate excipients list
DailyMed Link:	lomitapide mesylate at DailyMed

Pharmacology for lomitapide mesylate

Drug Class	Microsomal Triglyceride Transfer Protein Inhibitor
Mechanism of Action	Cytochrome P450 3A4 Inhibitors Microsomal Triglyceride Transfer Protein Inhibitors P-Glycoprotein Inhibitors

Anatomical Therapeutic Chemical (ATC) Classes for lomitapide mesylate

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

US Patents and Regulatory Information for lomitapide mesylate

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Chiesi	JUXTAPID	lomitapide mesylate	CAPSULE;ORAL	203858-004	Apr 23, 2015		RX	Yes	No	9,364,470
Chiesi	JUXTAPID	lomitapide mesylate	CAPSULE;ORAL	203858-004	Apr 23, 2015		RX	Yes	No	7,932,268
Chiesi	JUXTAPID	lomitapide mesylate	CAPSULE;ORAL	203858-002	Dec 21, 2012		RX	Yes	No	9,861,622
Chiesi	JUXTAPID	lomitapide mesylate	CAPSULE;ORAL	203858-001	Dec 21, 2012		RX	Yes	No	9,364,470
Chiesi	JUXTAPID	lomitapide mesylate	CAPSULE;ORAL	203858-006	Apr 23, 2015		DISCN	Yes	No	9,265,758

Expired US Patents for lomitapide mesylate

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Chiesi	JUXTAPID	lomitapide mesylate	CAPSULE;ORAL	203858-004	Apr 23, 2015	9,433,617
Chiesi	JUXTAPID	lomitapide mesylate	CAPSULE;ORAL	203858-002	Dec 21, 2012	9,861,622
Chiesi	JUXTAPID	lomitapide mesylate	CAPSULE;ORAL	203858-006	Apr 23, 2015	5,712,279
Chiesi	JUXTAPID	lomitapide mesylate	CAPSULE;ORAL	203858-001	Dec 21, 2012	9,433,617
Chiesi	JUXTAPID	lomitapide mesylate	CAPSULE;ORAL	203858-001	Dec 21, 2012	9,265,758

International Patents for lomitapide mesylate

Country	Patent Number	Title	Estimated Expiration
New Zealand	549721	Methods for treating disorders or diseases associated with hyperlipidemia and hypercholesterolemia while minimizing side-effects
South Korea	20060129082	METHODS FOR TREATING DISORDERS OR DISEASES ASSOCIATED WITH HYPERLIPIDEMIA AND HYPERCHOLESTEROLEMIA WHILE MINIMIZING SIDE-EFFECTS
Denmark	1725234
Spain	2399721
Japan	2016135762	高脂血症および高コレステロール血症に関連する障害または疾患を、副作用を最小限にしつつ処置するための方法 (METHODS FOR TREATING DISORDERS OR DISEASES ASSOCIATED WITH HYPERLIPIDEMIA AND HYPERCHOLESTEROLEMIA WHILE MINIMIZING SIDE-EFFECTS)

Supplementary Protection Certificates for lomitapide mesylate

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1725234	2013/063	Ireland		PRODUCT NAME: LOMITAPIDE OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; REGISTRATION NO/DATE: EU/1/13/851/001-003 20130731
1725234	92349	Luxembourg		PRODUCT NAME: LOMITAPIDE ET TOUTES SES FORMES THERAPEUTIQUEMENT EQUIVALENTES TELLES QUE PROTEGEES PAR LE BREVET DE BASE. FIRST REGISTRATION: 20130805
1725234	CR 2014 00002	Denmark		PRODUCT NAME: LOMITAPID ELLER ET FARMACEUTISK ACCEPTABELT SALT DERAF ELLER PIPERIDIN N-OXIDET DERAF, HERUNDER LOMITAPIDMESYLAT; REG. NO/DATE: EU/1/13/851/001-003 20130805
1725234	C300634	Netherlands		PRODUCT NAME: LOMITAPIDE EN ELKE THERAPEUTISCH EQUIVALENTE VORM DAARVAN, ZOALS DOOR HET BASISOCTROOI BESCHERMD; REGISTRATION NO/DATE: EU/1/13/851/001-003 20130731
1725234	C 2014 003	Romania		PRODUCT NAME: LOMITAPIDA SAU O SARE ACCEPTABILA FARMACEUTIC SAU PIPERIDIN-N-OXIDUL ACESTEIA; NATIONAL AUTHORISATION NUMBER: EU/1/13/851/001-003; DATE OF NATIONAL AUTHORISATION: 20130731; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/13/851/001-003; DATE OF FIRST AUTHORISATION IN EEA: 20130731

Market Dynamics and Financial Trajectory for Lomitapide Mesylate

Introduction

Lomitapide mesylate, marketed under the brand name Juxtapid, is a groundbreaking medication approved for the treatment of homozygous familial hypercholesterolemia (HoFH), a rare and life-threatening genetic disorder characterized by extremely high levels of low-density lipoprotein cholesterol (LDL-C). Here, we delve into the market dynamics and financial trajectory of this critical drug.

Clinical Indication and Usage

Lomitapide mesylate is indicated as an adjunct to a low-fat diet and other lipid-lowering treatments, with or without low-density lipoprotein apheresis, to reduce LDL-C, total cholesterol, apolipoprotein B, and triglycerides in patients with HoFH[1][2][5].

Market Need and Target Population

HoFH is a rare condition, affecting approximately 1 in 1 million people worldwide. Despite its rarity, the condition poses significant health risks, including premature cardiovascular disease. The market need for effective treatments is high, and lomitapide mesylate fills a critical gap in the therapeutic arsenal for managing HoFH[2][5].

Regulatory Approval and Orphan Drug Status

Lomitapide mesylate received FDA approval in 2012 and was granted orphan drug designation in 2007 due to its potential to treat a rare and serious condition. This designation provides various incentives, including market exclusivity and tax credits, which can significantly impact the drug's financial trajectory[1].

Pharmacoeconomic Analysis

The cost-consequence analysis (CCA) submitted by the manufacturer highlights the significant financial implications of lomitapide treatment. Over a five-year time horizon, the total cost of lomitapide added to standard care is estimated to be $1,503,014 per patient, compared to $70,392 for standard care alone. This translates to an annual incremental cost of $295,793 per patient[2][5].

Cost-Effectiveness

While lomitapide significantly reduces cardiovascular event risks (estimated reductions range from 45% to 57%), the cost-effectiveness is a subject of debate. The incremental cost-effectiveness ratios range widely, from $13.5 million per coronary procedure avoided to $512 million per cerebrovascular event avoided. These figures are subject to interpretation due to the uncertainty in the relationship between LDL-C reduction and clinical outcomes[5].

Patient Compliance and Discontinuation

The analysis assumes a 21% treatment discontinuation rate in the first year, with no discontinuation in subsequent years. Patients who discontinue treatment incur half a year’s cost of lomitapide and half a year’s treatment benefit in the year of discontinuation. Compliance rates are assumed to be 88% in the first year and 100% in subsequent years[5].

Safety and Monitoring Costs

Lomitapide is associated with several serious risks, including hepatic transaminase elevations, hepatic steatosis, small bowel and hepatic malignancies, and teratogenicity. These risks necessitate long-term monitoring, which adds to the overall cost of treatment. The pharmacovigilance program includes a 10-year observational study to gather data on these risks, further impacting the financial trajectory[3][4].

Market Size and Patient Population

Given the rarity of HoFH, the market size for lomitapide mesylate is relatively small. However, the high cost per patient and the extended treatment duration make it a significant market. The manufacturer estimates that a small cohort of patients (e.g., 11 patients) can result in cumulative costs of $16,533,156 over five years[5].

Competitive Landscape

Lomitapide mesylate is one of the few treatments available for HoFH, making it a critical component of the therapeutic regimen. The lack of direct competitors in this niche market allows lomitapide to maintain a strong market position, although the high cost and associated risks may limit its adoption in some healthcare systems[2].

Financial Projections

The annual cost of lomitapide treatment per patient is estimated at $300,602, with an incremental cost of $286,524 compared to standard care alone. Over a five-year period, the total cost per patient is projected to be $1,503,014. These costs are substantial and highlight the financial burden associated with treating HoFH with lomitapide mesylate[5].

Conclusion

Lomitapide mesylate is a crucial treatment for patients with HoFH, offering significant reductions in LDL-C and cardiovascular event risks. However, its high cost and associated safety risks make it a complex and costly therapeutic option. The financial trajectory of lomitapide is marked by high annual and cumulative costs, which are balanced against the potential for improved clinical outcomes.

Key Takeaways

• High Treatment Costs: Lomitapide treatment is associated with significant annual and cumulative costs.
• Clinical Effectiveness: The drug reduces LDL-C and cardiovascular event risks, but the cost-effectiveness is uncertain.
• Safety Monitoring: Long-term monitoring is required due to potential serious risks.
• Market Size: The market is small due to the rarity of HoFH, but the high cost per patient makes it significant.
• Competitive Position: Lomitapide maintains a strong market position due to the lack of direct competitors.

FAQs

Q: What is the primary indication for lomitapide mesylate? A: Lomitapide mesylate is indicated for the treatment of homozygous familial hypercholesterolemia (HoFH) as an adjunct to a low-fat diet and other lipid-lowering treatments.

Q: How much does lomitapide treatment cost per patient annually? A: The estimated annual cost of lomitapide treatment per patient is $300,602.

Q: What are the potential serious risks associated with lomitapide mesylate? A: The drug is associated with risks such as hepatic transaminase elevations, hepatic steatosis, small bowel and hepatic malignancies, and teratogenicity.

Q: How does lomitapide mesylate impact cardiovascular event risks? A: Lomitapide reduces cardiovascular event risks by 45% to 57% compared to standard care alone.

Q: What is the duration of the pharmacovigilance program for lomitapide mesylate? A: The pharmacovigilance program includes a 10-year observational study to gather data on the long-term risks associated with lomitapide use.

Sources

1. FDA Document: "203858Orig1s000ClinpharmR.pdf" - accessdata.fda.gov
2. NCBI Bookshelf: "Lomitapide (Juxtapid)" - ncbi.nlm.nih.gov
3. Pharmaceuticals.gov.in: "An Analysis on leveraging the patent cliff with drug sales worth USD 251 billion" - pharmaceuticals.gov.in
4. FDA Document: "203858Orig1s000OtherR.pdf" - accessdata.fda.gov
5. CDR Pharmacoeconomic Review Report: "Juxtapid PE Report" - cda-amc.ca