Last updated: February 22, 2026
What is NDC 33342-0277?
NDC 33342-0277 refers to Lumasiran, marketed under the brand name Oxlumo by Alnylam Pharmaceuticals. It is a gene-silencing therapy approved in 2020 for treating primary hyperoxaluria type 1 (PH1), a rare genetic disorder causing oxalate overproduction leading to kidney stones and renal failure.
Market Overview
Market Size and Incidence
- Target Population: Patients with primary hyperoxaluria type 1.
- Incidence rate: Estimated at 1 in 58,000 to 1 in 106,000 in the U.S.[1]
- Prevalence: Approximately 500 to 1,000 patients in the U.S., with similar proportions globally.
Competitive Landscape
- Existing treatments: Supportive care including hydration, citrate therapy, and dialysis.
- Competitors: Limited, but emerging gene therapies and enzyme replacement therapies are under development.
- Unique Value: First RNA interference (RNAi) therapy approved for this indication.
Market Dynamics
- The orphan drug designation confers market exclusivity until 2030.
- High unmet need drives willingness to pay.
- Adoption depends on clinician awareness, reimbursement policies, and demonstration of long-term efficacy.
Price Analysis
Current Pricing
- List Price (U.S.): Approximately $450,000 per patient annually.[2]
- Pricing Rationale: Reflects rarity, value based on clinical benefit, and innovation status.
Reimbursement Policies
- Secured via specialty pharmacy channels.
- Coverage considered favorable under most commercial insurers; Medicaid reimbursement varies regionally.
Cost-Effectiveness
- Cost-effectiveness is likely high given limited competition and the clinical need; incremental cost-effectiveness ratio (ICER) likely exceeds $150,000 per quality-adjusted life year (QALY) in absence of long-term outcomes.
Market Penetration and Forecasts
Adoption Rates
- Approved in multiple markets: U.S., EU, Japan.
- Estimated to treat approximately 20-30% of eligible patients in the first five years post-launch, due to rarity and limited awareness.
Revenue Projections (2023–2030)
| Year |
Estimated Patients Treated |
Revenue (USD millions) |
Assumptions |
| 2023 |
100 |
45 |
Initial adoption, slow uptake |
| 2025 |
300 |
135 |
Increased awareness, expanded reimbursement |
| 2030 |
700 |
315 |
Broader adoption, geographic expansion |
Projection assumes stable list price and no major price reductions.
Risks to Market Growth
- Emerging therapies potentially reducing demand.
- Price negotiations leading to discounts.
- Extended indication approvals or label changes.
Key Influencing Factors
- Regulatory updates: Expansions or label restrictions.
- Pricing negotiations: International price adjustments.
- Long-term efficacy data: Impact on affordability and late adopters.
- Market access policies: Reimbursement shifts.
Key Takeaways
- NDC 33342-0277 (Lumasiran/Oxlumo) has a specialized market with limited patient populations but high unmet needs.
- Current U.S. list price approximates $450,000 annually.
- Revenue forecasts suggest growth through expanded indications and geographic reach, reaching approximately $315 million globally by 2030.
- Market risks include emerging therapies, pricing pressures, and policy environment changes.
FAQs
1. How does Lumasiran compare price-wise to similar orphan drugs?
Orphan drugs for rare genetic disorders typically range from $200,000 to $500,000 annually; Lumasiran's price is near the upper end but justified by its novel mechanism.
2. What factors could influence price reductions?
Reimbursement negotiations, competitive entries, or data indicating long-term efficacy could pressure prices downward.
3. Is there potential for expanded indications?
Yes. Ongoing studies could lead to broader use in related oxaluria types, impacting revenue estimates.
4. How does market access differ internationally?
Pricing and reimbursement vary; European countries often negotiate lower prices, affecting global revenue consolidation.
5. What is the outlook for new entrants targeting primary hyperoxaluria?
Development of alternative gene therapies or enzyme replacement options could fragment the market, impacting Lumasiran's share.
References
[1] Dally, M., et al. (2019). Epidemiology of Primary Hyperoxaluria. European Journal of Clinical Investigation.
[2] Alnylam Pharmaceuticals. (2022). Oxlumo Pricing and Reimbursement Details. Official Release.
