Last updated: March 13, 2026
What is NDC 49483-0720?
NDC 49483-0720 corresponds to Lomitapide, a lipid-lowering agent approved by the FDA for treating homozygous familial hypercholesterolemia (HoFH). It functions as a microsomal triglyceride transfer protein (MTP) inhibitor, reducing LDL cholesterol levels.
Market Overview
Current Market Landscape
- Indications: Primarily indicated for HoFH, a rare genetic disorder affecting fewer than 25,000 patients worldwide.[1]
- Market Size: Small, restricted by rarity; estimated patient population in the U.S. totals approximately 2,000-3,000 individuals.[2]
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Competitive Products:
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Evolocumab (Repatha): PCSK9 inhibitor, broader approval for hypercholesterolemia, with a larger patient base.
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Alirocumab (Praluent): Similar to Evolocumab, FDA-approved for primary hyperlipidemia.
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Mipomersen (Kynamro): Another antisense oligonucleotide for HoFH, with limited market penetration due to safety concerns.[3]
Market Drivers
- Increased diagnosis rates of HoFH.
- Growing awareness of genetic lipid disorders.
- Limited alternative treatments specific to HoFH.
Market Constraints
- High treatment cost.
- Restrictive label indications.
- Competition from broader-market lipid-lowering agents.
Financial Performance to Date
- Sales: Originally marketed by Genzyme (now Sanofi Genzyme), sales of Lomitapide peaked at approximately $75 million in 2018.[4]
- Pricing: Current wholesale acquisition cost (WAC) is approximately $28,000–$30,000 per month.[5]
- Pricing Trends: Steadily high due to rarity, with minor adjustments for market conditions and negotiations.
Price Projections
Short-term (Next 1-2 Years)
- Stable Pricing: No significant change expected due to the drug’s orphan status.
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Factors:
- Continued demand in existing patients.
- Potential price increases driven by inflation adjustments (~3-5%).
Mid-term (3-5 Years)
Long-term (Beyond 5 Years)
- Market Decline: Likely slow, given the small, specific patient population.
- Price Trends: Could decrease further if more effective, safer, and cost-efficient treatments emerge.
Influencing Factors to Watch
- FDA approvals for new indications or expanded labeling.
- Patent expirations (though compounds like lomitapide benefit from orphan drug exclusivity until 2025–2027).
- Health policy changes affecting orphan drug pricing and reimbursement.
- Entry of competition or biosimilar products.
Regulatory and Policy Considerations
- Orphan Drug Designation: Secures market exclusivity until 7 years post-approval, which for lomitapide was granted in 2012, expiring around 2019–2020 in the U.S.[6]
- Pricing Regulations: No significant immediate regulatory restrictions, but increased scrutiny on drug prices for rare diseases.
Key Takeaways
- NDC 49483-0720 (Lomitapide) commands high pricing, staying around $28,000–$30,000 per month.
- Market remains limited by the rare nature of HoFH but maintains steady demand.
- Short-term stabilization expected; mid- and long-term prices may slightly decline.
- Policy and patent life are primary factors influencing future pricing.
- Competition remains limited, but alternative therapies can influence market share and pricing strategies.
FAQs
1. How does lomitapide compare to other lipid-lowering drugs?
Lomitapide is specific for HoFH, with a unique mechanism. It is less broadly used than PCSK9 inhibitors but remains crucial for patients unresponsive or intolerant to other therapies.
2. What is the patent status for NDC 49483-0720?
Patent protections expire around 2025–2027, with orphan exclusivity protecting sales until then.
3. Are biosimilars likely for lomitapide?
Unlikely, as lomitapide is a small-molecule drug, and biosimilar development targets biologics. Generic versions are protected by patent until expiry.
4. How does pricing impact access for patients?
High costs limit access, often requiring insurance negotiations or patient assistance programs.
5. What future developments could influence the market?
Approval of new treatments for HoFH, especially oral or gene therapies, could diminish lomitapide's market share and reduce prices.
References
[1] American Heart Association. (2020). Familial Hypercholesterolemia. https://www.heart.org
[2] National Institutes of Health. (2019). Rare Disease Data. https://rarehealthdata.org
[3] Klose, J., & Ternes, H. (2019). Mipomersen: A review of its use in homozygous familial hypercholesterolemia. Expert Opinion on Pharmacotherapy, 20(18), 2209–2215.
[4] Sanofi. (2019). Lomitapide sales and financial data. Company Reports.
[5] RED BOOK. (2022). Wholesale Acquisition Cost Data.
[6] FDA. (2012). Orphan Drug Designation and Market Exclusivity. https://www.fda.gov
