Last updated: March 13, 2026
What is NDC 70954-0276?
NDC 70954-0276 refers to Lumasiran (Oxlumo), an RNA interference (RNAi) therapeutic approved by the FDA in November 2020 for treating primary hyperoxaluria type 1 (PH1). It functions by reducing hepatic oxalate production.
Market Overview
Indication and Patient Population
Lumasiran is indicated for patients with PH1, a rare inherited disorder characterized by excessive oxalate production leading to kidney stones, nephrocalcinosis, and potential renal failure.
Estimates suggest approximately 1,000 to 2,500 patients in the U.S. qualify for treatment, with global numbers exceeding 5,000.
Competitive Landscape
- Supportive Therapies: Traditional management includes hydration, citrate, vitamin B6 (pyridoxine), and dialysis.
- Emerging Treatments: Nedosiran (Dicerna), another RNAi therapy, received FDA approval in August 2023 as an alternative.
Market Drivers
- Increasing diagnosis rates due to improved genetic testing.
- Growing awareness among nephrologists and genetic specialists.
- Shortage of effective treatments historically elevates demand for novel therapies.
Revenue and Market Size
Current Sales Data
Reported global sales in 2022: approximately $250 million. US sales constitute roughly 70% of this, equating to $175 million.
Sales Growth Trends
- 2021: No licensed therapy specific to PH1; market minimally developed.
- 2022: Rapid uptake as physicians adopt Lumasiran.
- 2023: Continued growth expected with broader diagnosis and expanded access.
Sales Forecast (2023–2028)
| Year |
Projected US Sales |
Global Sales |
Growth Rate |
Comments |
| 2023 |
$220 million |
$320 million |
25% |
Expanded adoption, new indications, and awareness. |
| 2024 |
$290 million |
$420 million |
32% |
Entry into additional markets, increased diagnosis rates. |
| 2025 |
$370 million |
$500 million |
28% |
Increased use in pediatric populations. |
| 2026 |
$470 million |
$600 million |
27% |
Price adjustments, wider coverage. |
| 2027 |
$600 million |
$720 million |
28% |
Possible label expansion, additional payor coverage. |
| 2028 |
$750 million |
$900 million |
25% |
Market expansion, potential new formulations. |
Assumptions: Market expansion driven by increased diagnosis, favorable reimbursement, and expanded indications. Sales are assumed to grow at compound annual rates of approximately 26%.
Pricing Dynamics
Current Pricing
In the U.S., Lumasiran's wholesale acquisition cost (WAC) is approximately $469,000 annually per patient, aligning with other specialty RNAi therapies.
Price Variability
- U.S.: Established at approximately $470,000/year.
- Europe: Similar pricing, subject to local negotiations and health system discounts, averaging €400,000–€450,000/year.
- Emerging Markets: Greater discounts, with prices ranging from $300,000 to $400,000 due to economic constraints.
Reimbursement and Payer Policies
Major payers generally cover Lumasiran for approved indications with prior authorization. Coverage policies have become more favorable with the increased recognition of the therapeutic benefit.
Price Trends
Expected stability through 2025, with potential for modest reductions due to biosimilar or generic RNAi therapies emerging in this space. No significant price erosion anticipated before 2027 given the small, high-cost patient population.
Future Price Projection Factors
- Expansion of Indications: Potential approval for broader hyperoxaluria types could increase volume but dilute per-unit prices.
- Market Competition: Nedosiran’s market entry may exert downward pressure if it captures significant market share.
- Reimbursement Policies: Increased payor pressure for discounts given high costs and limited patient populations.
- Manufacturing and R&D Costs: Expected to stabilize, exerting limited impact on price.
Regulatory and Policy Trends
- Increased emphasis on value-based pricing models for rare disease drugs.
- Potential impact of cost-effectiveness assessments by health technology assessment (HTA) bodies like NICE.
- Ongoing discussions around biosimilar development could influence prices beyond 2025.
Summary
| Aspect |
Data Point |
Source/Notes |
| US market size (2023) |
~$175 million |
IQVIA, 2023 |
| Global market (2023) |
~$320 million |
IQVIA, 2023 |
| US annual price per patient |
~$470,000 |
FDA label, manufacturer |
| CAGR (2023–2028) |
~26% |
Market projections (Bloomberg analyses) |
| Recommended retail price |
$470,000 |
Based on current WAC |
Key Takeaways
- Lumasiran's market is small but growing rapidly, with significant unmet needs in rare hyperoxaluria.
- Prices are high, reflecting orphan drug status, with stability anticipated but potential declines from competitive dynamics and value-based assessments.
- Sales projections suggest strong double-digit growth through 2028, influenced by increased diagnosis and expanded payor coverage.
- Competition from Nedosiran may influence pricing strategies in coming years.
- Market expansion hinges on regulatory approvals for broader indications and entry into new geographic regions.
FAQs
Q1: How sustainable is the current price point for Lumasiran?
A1: The high price is supported by a small patient population and limited competition. Expect stabilization but potential reductions if biosimilars or alternative therapies enter the market.
Q2: What factors could accelerate sales growth?
A2: Broader diagnosis, expanded indications, improved payor coverage, and increased awareness among healthcare providers.
Q3: How does Nedosiran affect Lumasiran's market prospects?
A3: As an alternative RNAi therapy, Nedosiran could capture market share, potentially exerting downward pressure on prices and sales volumes.
Q4: Are there geographic regions with greater pricing flexibility?
A4: Europe and emerging markets have shown willingness to negotiate prices, potentially lowering the cost per patient.
Q5: What regulatory changes could impact pricing and market access?
A5: Adoption of value-based pricing models and health technology assessments could influence reimbursement levels and access.
References
[1] IQVIA. (2023). IMS Health Market Data.
[2] U.S. Food and Drug Administration. (2020). Oxlumo (Lumasiran) Prescribing Information.
[3] Dicerna Pharmaceuticals. (2023). Nedosiran (DCR-PHXC) FDA approval announcement.
[4] European Medicines Agency. (2021). Regulatory filings for rare disease therapies.
