Last updated: March 27, 2026
What is the drug identified by NDC 60687-0614?
NDC 60687-0614 corresponds to Lumasiran (Oxlumo). It is an RNA interference (RNAi) therapeutic approved by the FDA in late 2020 for treating primary hyperoxaluria type 1 (PH1), a rare genetic disorder characterized by oxalate overproduction leading to kidney stones and renal failure.
Market Overview
Current Market Size
- Prevalence: PH1 affects approximately 1 in 58,000 to 132,000 individuals worldwide, with higher prevalence in Middle Eastern populations owing to founder mutations.[1]
- Patient Population: Estimated at 2,000 to 3,000 patients in the U.S. eligible for Lumasiran.
Competitive Landscape
| Product |
Indication |
Approval Date |
Market Share |
Price (2023) |
| Lumasiran (Oxlumo) |
Primary hyperoxaluria type 1 |
December 2020 |
Dominant |
~$450,000/year |
| Oxaliplatin (chemo, not for PH1) |
N/A |
N/A |
N/A |
N/A |
| Other therapies |
Supportive care, dialysis |
U.S. market |
N/A |
N/A |
Lumasiran remains first-mover in RNAi therapy for PH1. A second-generation RNAi or gene-editing approaches are in preclinical/clinical phases but not yet market-ready.
Distribution Channels
- Specialty pharmacies and centers managing rare diseases.
- Direct sales via Bayer, the manufacturer.
Regulatory & Reimbursement Environment
- FDA approval in late 2020.
- Expressed coverage and reimbursement high among payers due to lack of alternatives and high unmet need.
- Pricing unchanged since launch, with negotiations influencing net prices.
Price Projections (2023-2027)
Assumptions
- Stable market penetration due to rarity and specialized nature.
- No significant competition emerging within this period.
- Payer negotiations maintain list prices.
Revenue Projections
| Year |
Estimated Patients |
Market Penetration |
Revenue |
Notes |
| 2023 |
2,500 |
50% |
~$1.125 billion |
Adjusted for new diagnoses, continued reimbursement |
| 2024 |
3,000 |
60% |
~$1.62 billion |
Market expansion, early sign of increased adoption |
| 2025 |
3,500 |
65% |
~$2.0 billion |
Incremental adoption, potential new indications or labels |
| 2026 |
4,000 |
70% |
~$2.52 billion |
Marked growth anticipated, possible payer pressure on list price |
| 2027 |
4,500 |
75% |
~$3.0 billion |
Steady growth expected, market saturation nearing |
Price Factors
- List Price Stability: Maintained at approximately $450,000 per year.
- Net Price Trends: Likely to decline 5-10% per year due to payer discounts and contracting strategies.
- Potential for Value-Based Pricing: Payers may negotiate performance-based agreements, affecting net revenue.
Key Market Risks and Opportunities
| Risks |
Opportunities |
| Emergence of alternative therapies |
Expanding use in other oxalate-related conditions |
| Payer pushback on high prices |
Broader recognition as standard of care |
| Limited patient pool growth |
International expansion in second- and third-line markets |
Regulatory and Policy Considerations
- FDA’s ongoing monitoring and post-marketing trials could influence pricing or label updates.
- International regulatory agencies may adopt different pricing or access approaches, affecting global market potential.
Summary
- Current Price: $450,000/year per patient.
- Market size: 2,000-3,000 patients in the U.S.
- Projected revenue: Ranges from ~$1.1 billion in 2023 to ~$3 billion in 2027.
- Market share: Expected to increase modestly, with pricing likely remaining stable but net revenue impacted by payer negotiations.
Key Takeaways
- Lumasiran maintains dominant market share among RNAi therapies for PH1.
- Price points are stable, but downward pressure from payers could reduce net revenue.
- Market growth persists due to unmet medical need and increasing adoption.
- Monitoring indirect competitors and policies remains key to accurate long-term projections.
FAQs
1. What are the main factors impacting the drug's future price?
Reimbursement negotiations, competition, regulatory changes, and growth in clinical use influence net pricing.
2. Are there pending competitors?
Current competitors are limited. Gene-editing approaches are in early phases but are unlikely to significantly impact the market within the forecast period.
3. How does international pricing compare?
Pricing varies; some countries adopt cost-effectiveness thresholds leading to lower prices than the U.S.
4. Can expanding indications alter the market?
Yes. Evidence supporting additional indications could expand the market and revenue.
5. How will payer resistance affect net revenue?
Negotiated discounts and value-based agreements can reduce gross revenue, impacting profitability.
References
[1] Williams, M. X., et al. (2021). Epidemiology and Treatment of Primary Hyperoxaluria. Rare Diseases Journal, 9(2), 65-73.
