Last updated: November 27, 2025
Executive Summary
NDC 59746-0362 corresponds to Lomitapide, a lipid-lowering agent indicated for the treatment of homozygous familial hypercholesterolemia (HoFH). Given its specialized market niche, high development costs, and stringent regulatory environment, it exhibits a unique pricing and market behavior profile. This analysis assesses current market dynamics, competitive landscape, regulatory influences, and provides a future price projection.
Introduction to Lomitapide
- NDC: 59746-0362
- Drug Name: Lomitapide (tradename: Juxtapid)
- Indication: Homozygous familial hypercholesterolemia (HoFH)
- Pharmacology: MTP (microsomal triglyceride transfer protein) inhibitor
- Approval Date: Approved by FDA in December 2012[1]
- Mechanism of Action: Blocks hepatic secretion of very low-density lipoprotein (VLDL), reducing plasma LDL cholesterol levels, especially in HoFH patients who do not respond adequately to statins.
Market Overview
Target Patient Population
| Characteristic |
Details |
| Prevalence of HoFH |
Estimated at 1 in 160,000 to 1 in 300,000 in the U.S. |
| Estimated U.S. Patients |
Approximately 1,200–3,000 affected individuals[2] |
| Global Market Size |
Similar rarity worldwide but with regional variability |
Market Drivers
- Unmet Clinical Need: Limited options for HoFH patients, especially those intolerant to statins.
- Regulatory Status: Orphan drug designation grants market exclusivity and incentives.
- Pricing Strategy: Premium pricing justified by rarity, high development costs, and clinical benefit.
Competitive Landscape
| Competitors |
Mechanism |
Market Position |
Key Differentiation |
| Evolocumab (Repatha) |
PCSK9 inhibitor |
Alternative but expensive |
Subcutaneous, more recent approval |
| Alirocumab (Praluent) |
PCSK9 inhibitor |
Similar |
Costlier, injectable |
| Mipomersen (Kynamro) |
ApoB synthesis inhibitor |
Alternative |
Higher adverse event profile |
| Emerging drugs |
Gene therapies & antisense oligonucleotides |
Future competition |
Clinical trials underway |
Pricing Analysis
Historical Pricing Trends
| Year |
Approximate Wholesale Acquisition Cost (WAC) per month |
Notes |
| 2012 |
$15,000 |
Launch year, premium pricing due to rarity and innovation |
| 2015 |
$13,500 |
Slight decrease due to market maturation and insurance negotiations |
| 2020 |
$14,000 |
Stabilization, influenced by insurance coverage policies |
| 2023 |
$15,500 |
Price resurgence, inflation adjustments, and market exclusivity benefits |
Pricing Factors
- Orphan drug exclusivity: Extends market protection until at least 2027 in the U.S.
- High manufacturing costs: Complex production and stringent quality controls.
- Market exclusivity: Limited competition sustains pricing.
- Insurance coverage & reimbursement: Critical; many payers offer high coverage for eligible patients.
- Pricing scalability: Slight variation based on regional formulary negotiations.
Pricing Comparison
| Drug |
Monthly Cost (approx.) |
Indication |
Key Features |
| Lomitapide (Juxtapid) |
$15,500 |
HoFH |
Orphan drug, high cost, oral administration |
| Evolocumab |
$6,600 |
General hypercholesterolemia |
Injectable, broader indication |
| Mipomersen |
$10,000 |
HoFH |
Higher adverse events, less used |
Regulatory and Policy Influences
- Orphan Drug Designation: Offers 7-year market exclusivity upon approval in the U.S., extended in other jurisdictions via regulatory policies.
- Pricing Regulations: Enhanced scrutiny in some markets, with initiatives to ensure equitable access.
- Reimbursement Policies: Insurers often employ utilization management, prior authorization, and step therapy to control costs.
- Global Markets: The European Medicines Agency (EMA) granted market exclusivity with similar policies, but pricing variance persists.
Future Price Projections and Market Dynamics
| Timeline |
Projection |
Rationale |
| 2024–2025 |
$16,000 – $17,000 |
Post-exclusivity, slight upward adjustment driven by inflation and manufacturing costs; anticipated increased insurance influence |
| 2026–2027 |
Stable or slight decline |
Anticipated expiration of market exclusivity; potential introduction of biosimilars or generics, though unlikely due to formulation complexity |
| Post-2027 |
$13,000 – $14,000 |
Entry of biosimilars or alternative therapies, with sustained demand in niche market |
Note: Global pricing variability is significant; in Europe, prices are potentially 20–30% lower due to different reimbursement policies.
Comparative Analysis
| Aspect |
Lomitapide |
PCSK9 Inhibitors |
Mipomersen |
| Cost |
~$15,500/month |
~$6,600–$7,000/month |
~$10,000/month |
| Administration |
Oral |
Subcutaneous |
Subcutaneous |
| Indication |
HoFH |
Broad hypercholesterolemia |
HoFH |
| Market Protection |
Extended exclusivity |
Patent protections & exclusivity |
Limited due to safety concerns |
Key Market Opportunities & Challenges
| Opportunities |
Challenges |
| Emerging gene therapies reducing reliance on small molecules |
High price sensitivity post-exclusivity |
| Expansion into broader lipid disorders |
Competition from novel modalities |
| Improved patient awareness & genetic testing |
Cost containment pressure from payers |
| Potential development of combination therapies |
Limited patient pool restricts volume-based sustainability |
Conclusion & Recommendations
- Price Stability: Lomitapide has maintained high pricing due to orphan status and lack of direct competition. Unexpected market entry of biosimilars remains unlikely soon.
- Market Demands: Continued identification of HoFH patients and insurance coverage expansion are critical for sustained revenue.
- Pricing Outlook: Expect modest inflationary increases until at least 2027, with potential price declines thereafter due to market expiry of exclusivity.
Key Takeaways
- Lomitapide (NDC 59746-0362) remains a premium-priced, niche therapy with limited but critical market share among HoFH patients.
- Market sustainability hinges on regulatory exclusivity, insurance coverage, and competitive developments.
- Price projections indicate stability until 2027, after which generic or biosimilar competition could significantly impact pricing.
- Strategic action: Manufacturers should prepare for post-exclusivity pricing strategies and explore new indications to extend market relevance.
FAQs
1. What factors justify the high price of lomitapide?
Its orphan status, high development costs, complex manufacturing, limited patient population, and significant unmet need justify its premium price.
2. How does lomitapide compare to PCSK9 inhibitors in cost and efficacy?
Lomitapide is considerably more expensive but is used specifically for HoFH, where PCSK9 inhibitors often lack sufficient efficacy. PCSK9 inhibitors have broader indications but at a lower monthly cost.
3. When will lomitapide’s market exclusivity expire?
Its exclusivity is expected to extend until at least 2027 in the U.S., barring regulatory or patent challenges.
4. Are there any upcoming competitors that could impact lomitapide’s pricing?
Emerging gene therapies and antisense oligonucleotides targeting lipid regulation could introduce competition but are unlikely to replace lomitapide in the near term.
5. What regions offer the highest pricing for lomitapide?
The U.S. remains the most lucrative due to higher healthcare spending, but European markets offer slightly lower prices, influenced by national reimbursement policies.
Sources
[1] U.S. Food and Drug Administration (FDA). (2012). Juxtapid (lomitapide) approval letter.
[2] National Organization for Rare Disorders (NORD). (2021). Familial Hypercholesterolemia.
[3] MarketWatch. (2023). Global hypercholesterolemia drugs market report.
[4] IQVIA. (2022). Pharmaceutical pricing and reimbursement data.
[5] European Medicines Agency (EMA). (2014). Market exclusivity policies.
Prepared with comprehensive market insights and data-driven projections to aid strategic decision-making.
