Last updated: February 26, 2026
Overview
The drug identified by NDC 42291-0347 corresponds to Lumasiran (Oxalosis and Primary Hyperoxaluria Type 1). It is marketed under the brand name Oxlumo, developed by Alnylam Pharmaceuticals. Approved by the FDA in November 2020, Lumasiran is the first RNA interference therapy targeting primary hyperoxaluria type 1 (PH1).
Market Landscape
Target Population
- PH1 is a rare genetic disorder characterized by overproduction of oxalate leading to kidney stones and progressive kidney damage.
- Prevalence estimates suggest approximately 1 in 1 million to 1 in 200,000 individuals in the U.S. are affected.
- Estimated total U.S. patient pool: approximately 300-400 patients.
Competitive Environment
- Primary competitors include older treatments like dialysis, kidney transplants, and supportive therapies.
- No direct RNAi competitors currently approved.
- Off-label use of metabolic disorder medications is limited.
Market Drivers
- Increasing awareness of PH1.
- Growing adoption of genetic therapies.
- Significant unmet medical needs among the patient population.
- Payer acceptance following FDA approval, driven by the drug’s targeted mechanism.
Regulatory and Reimbursement Factors
- FDA approval granted based on Phase 3 clinical trial outcomes showing significant reduction in urinary oxalate.
- Insurance coverage is evolving, with payers increasingly favoring targeted therapies for rare diseases, leading to premium pricing strategies.
Price Analysis
Current Pricing Benchmark
- List price of Oxlumo: approximately $450,000 annually per patient in the U.S. (as of Q4 2022).
- Pricing is aligned with other gene-based therapies, factoring the high development costs and rarity of disease.
Cost Components
- Drug acquisition cost: $450,000/year.
- Administration and monitoring costs: approximately $20,000 annually.
- Total annual treatment cost: ~$470,000.
Price Projections
| Year |
Scenario |
Estimated Price Range |
Comment |
| 2023 |
Baseline |
$450,000 |
Current list price, with potential for slight increases due to inflation and increased demand. |
| 2024 |
Moderate increase |
$470,000 - $490,000 |
Anticipated due to increased market penetration and expansion of payer coverage. |
| 2025 |
Stabilization |
$490,000 - $510,000 |
Expected after initial adoption phase, assuming no significant price reductions. |
| 2026+ |
Cost containment |
$470,000 - $500,000 |
Potential discounts, longer-term negotiations, or biosimilar entry may influence pricing. |
Future Market and Price Trends
- Market Expansion: Broader application or label expansion could increase patient numbers.
- Pricing Pressure: Payer pushback on high-cost rare disease drugs may result in rebates/discounts.
- Biosimilar Entry: No biosimilars are currently in development; entry is unlikely before 2028.
- Reimbursement Landscape: Growth in managed care coverage may moderate annual price increases.
Key Market Barriers
- Small patient population limits revenue potential.
- High development costs are offset by premium pricing strategies.
- Potential regulatory or reimbursement changes affecting drug profitability.
Key Takeaways
- NDC 42291-0347 (Lumasiran/Oxlumo) has a clinical monopoly in primary hyperoxaluria type 1 treatment.
- Current list price is approximately $450,000 per year, with projections indicating modest increases over time.
- Market expansion depends on label expansion and increased awareness.
- Reimbursement pressures and healthcare cost containment efforts may influence future pricing.
- The small target population constrains revenue, but high unmet need supports premium pricing.
FAQs
What is the main competitive advantage of Lumasiran?
It is the first FDA-approved RNA interference therapy specifically for PH1, offering targeted treatment with significant reductions in urinary oxalate.
How does the price of Oxlumo compare to other rare disease treatments?
It is comparable or slightly higher than similar gene therapies, often exceeding $400,000 annually due to the high cost of development and small patient populations.
Are biosimilars or generics expected for Lumasiran?
No biosimilars are in development currently; biosimilar entry is unlikely before 2028 given regulatory and development timelines.
What factors could influence future pricing?
Increased payer pressure, expanded indications, or biosimilar competition could lead to price reductions or rebates.
What is the estimated market size for this drug?
Approximately 300-400 patients in the U.S., translating to a potential annual revenue of around $135 million to $180 million at current prices.
References
[1] Alnylam Pharmaceuticals. (2022). Oxlumo (lumasiran) prescribing information. Retrieved from https://www.alnylam.com
[2] U.S. Food and Drug Administration. (2020). FDA approves novel therapy for rare kidney disease. Retrieved from https://www.fda.gov
[3] IQVIA. (2022). Rare Disease Market Trends Report.
[4] GlobalData. (2022). RNA Interference Therapeutics Market Analysis.
