Last updated: February 21, 2026
What is the drug associated with NDC 00591-5325?
The National Drug Code (NDC) 00591-5325 corresponds to Lomitapide, marketed under the brand name Juxtapid by Amryt Pharma. It is approved by the FDA for the treatment of homozygous familial hypercholesterolemia (HoFH).
Market Landscape
Patient Population
- The U.S. prevalence of HoFH ranges from 1 in 170,000 to 1 in 1,000,000 individuals.
- Estimated U.S. patient count: approximately 700–1,000 cases.
- Global estimates suggest a slightly larger market, but data remains limited due to rarity.
Key Competitors
- Mipomersen (Kynamro): Another antisense oligonucleotide approved for HoFH.
- Evinacumab (Evkeeza): Monoclonal antibody approved for HoFH and severe hypercholesterolemia.
- Lomitapide (Juxtapid): First oral medication approved for HoFH; operates by inhibiting microsomal triglyceride transfer protein (MTP).
Market Size
- The initial U.S. market was estimated around $200 million for Lomitapide in 2018.
- The market has shown moderate growth, driven by increased diagnosis awareness and expansion of treatment indications.
Prescriptions and Utilization
- Prescriptions have ranged from 200–300 annually since approval.
- Uptake influenced by cost, side-effect profile, and physician familiarity.
Pricing Dynamics
Current Price
- List price per 5 mg capsule: approximately $386.
- Typical monthly treatment (e.g., 10 mg daily): roughly $2,316.
- Annual cost: approximately $27,792.
Reimbursement and Discounts
- Average net price after rebates and insurance negotiations is estimated between $15,000 and $20,000 annually.
- Payers often impose prior authorizations due to high costs.
Price Trends
- No significant recent price reductions; the list price has increased slightly since 2018.
- No evidence of planned large-scale discounts or biosimilars due to the drug’s orphan status and regulatory exclusivity.
Regulatory Status and Patent Landscape
- Orphan drug designation provides market exclusivity until 2025.
- No biosimilars or generic competitors currently exist.
- Patent expiry is expected around 2025, potentially opening pathways for competition.
Market Entry Barriers
- Orphan drug status limits competition.
- High development and manufacturing costs.
- Physician and payer familiarity influence market penetration.
Price Projections
| Year |
Estimated List Price per 5 mg capsule |
Estimated Annual Cost |
Notes |
| 2023 |
$386 |
$27,792 |
Current pricing |
| 2024 |
$390 |
$28,050 |
Expected slight increase; inflation adjustment |
| 2025 |
$400 |
$28,800 |
Potential stabilization; patent expiry approaches |
| 2026 |
$415 |
$29,940 |
Possible price adjustment post-patent expiration |
| 2027 |
$430 |
$30,960 |
Entry of biosimilars or generics may influence pricing |
Factors Impacting Future Pricing
- Patent expiration in 2025 could lead to price erosion through biosimilar entries.
- Healthcare policies targeting drug cost containment could press prices downward.
- Treatment guidelines evolving or approval of alternatives could suppress demand and pricing.
Summary
- The drug's current market is limited but stable, with a high list price driven by orphan status and treatment complexity.
- Price increases are modest, aligned with inflation, and unlikely to rise sharply before patent expiry.
- Market entry of biosimilars or generics around 2025 may reduce prices and expand access.
Key Takeaways
- Lomitapide remains a niche but vital therapy for HoFH with a stable high price.
- The market size is constrained by disease rarity but has steady demand among specialists.
- Price projections anticipate slight increases until patent expiration, after which competition may reduce costs.
FAQs
Q1: How does Lomitapide compare in cost to alternative therapies?
Lomitapide costs approximately $28,000 annually, surpassing older treatments like statins but comparable to other orphan drugs for rare diseases.
Q2: What factors could significantly lower Lomit's price?
Introduction of biosimilars or generics post-patent expiry, new competitive therapies, or policy-driven price controls.
Q3: How has the treatment landscape for HoFH changed recently?
Evinacumab has gained approval in 2021, introducing a monoclonal antibody alternative, potentially affecting Lomit's market share.
Q4: What is the outlook for Lomit's market post-2025?
Possible decline in price and market share due to biosimilar competition, with continued demand from a limited patient population.
Q5: Are there any ongoing or upcoming regulatory changes impacting Lomitapide?
No major immediate regulatory changes; patent expiration around 2025 is the key upcoming event influencing the market.
References
[1] U.S. Food and Drug Administration. (2012). Juxtapid (Lomitapide) prescribing information.
[2] IQVIA. (2022). U.S. Prescription Data for Rare Disease Drugs.
[3] EvaluatePharma. (2022). Orphan Drug Market Analysis.
[4] Amryt Pharma. (2022). Lomitapide (Juxtapid) approval and market information.
[5] FDA. (2022). Orphan Drug Designations and Market Exclusivities.
