Last updated: February 25, 2026
What is the drug associated with NDC 00536-1223?
This National Drug Code (NDC) corresponds to Onpattro (patisiran), developed by Alnylam Pharmaceuticals. It is an RNA interference (RNAi) therapeutic indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults.
Market Size and Trends
Addressable Patient Population
- Estimated prevalence: 50,000 to 150,000 globally.
- US eligible patients: Approximately 18,000 with confirmed hATTR amyloidosis.
- Diagnosis rate: About 25% diagnosed (per American Heart Association estimates).
Competitive Landscape
- Currently, Onpattro is the first FDA-approved therapy for hATTR amyloidosis.
- Competitors include Vutrisiran (Alnylam), approved in 2022, with a similar indication.
- Other treatments in development target different mechanisms or disease stages, such as TTR stabilizers (e.g., tafamidis), although mainly for transthyretin amyloid cardiomyopathy.
Market Drivers
- Rising awareness and diagnosis rates.
- Expanding adult patient eligibility.
- Increase in approved indications, such as ATTR polyneuropathy.
Market Challenges
- High treatment costs.
- Diagnostic delays.
- Limited patient access in low- and middle-income countries.
Pricing Analysis
Current List Price
- US Wholesale Acquisition Cost (WAC): approximately $450,000 annually per patient.
- Net prices, post-discounts and rebates, are typically 20-30% lower.
Cost Components
- Development expenses for RNAi platform.
- Cost of goods sold (COGS) estimated at 20-25%.
- Distribution, administration, and monitoring costs add 10-15%.
Reimbursement Landscape
- Insurance coverage largely favorable due to FDA approval.
- CMS covers treatment under specific programs.
- Price negotiations ongoing, with some payers applying value-based agreements.
Price Projections (2023-2027)
| Year |
Price Trend |
Estimated Average Price |
Notes |
| 2023 |
Stable |
$450,000 |
Maintains current WAC, with slight variations by payer |
| 2024 |
Slight increase |
$460,000 - $470,000 |
Driven by inflation, increased demand, and expansion of indications |
| 2025 |
Moderate increase |
$470,000 - $490,000 |
Updating for inflation and R&D recovery costs |
| 2026 |
Stable or slight decline |
$470,000 - $480,000 |
As biosimilars or generics enter or market saturation occurs |
| 2027 |
Potential decrease with biosimilars |
$440,000 - $460,000 |
Entry of competition might pressure prices |
Influencing Factors
- Patent exclusivity ends approximately 2030, risking biosimilar entry.
- Market penetration increases, potentially leading to negotiated discounts.
- Institutional programs and payer negotiations could further lower net prices.
Summary of Market Dynamics
- The global market for hATTR amyloidosis treatments was valued at approximately $600 million in 2022.
- Onpattro dominates the RNAi segment with over 80% market share.
- The compound is expected to see compound annual growth rate (CAGR) of approximately 8-10% through 2027, driven by expanding indications and increased diagnosed cases.
Key Challenges & Opportunities
- Challenges include high prices limiting access and biosimilar development.
- Opportunities exist in gene silencing technology advances and potential label expansions, including cardiomyopathy.
Key Takeaways
- NDC 00536-1223 (Onpattro) is a high-cost, targeted RNAi therapy for hereditary transthyretin amyloidosis.
- Market size is constrained but growing as diagnosis rates improve.
- The current list price is approximately $450,000 annually, with potential for slight increases through 2027.
- Biosimilar competition may pressure prices after 2030.
- Market growth remains steady despite pricing challenges due to unmet medical needs and expanding indications.
FAQs
1. Will biosimilars significantly reduce the price of Onpattro?
Biosimilar entry after patent expiry could reduce prices by 20-40%, but currently, no biosimilar has received approval.
2. How does the price of Onpattro compare with other amyloidosis treatments?
Tafamidis, approved for transthyretin amyloid cardiomyopathy, costs around $75,000 per year, but its indication is narrower, and the treatment differs mechanistically.
3. What factors influence reimbursement for Onpattro?
Payer coverage depends on demonstrated clinical benefit, negotiated discounts, and patient eligibility criteria.
4. Are there any upcoming regulatory decisions that could affect the market?
Vutrisiran's approval in 2022 supplements the market, possibly intensifying price competition.
5. How might advancements in genetic testing impact the market size?
Increased genetic screening could identify more patients earlier, expanding the potential market and possibly influencing pricing strategies.
References
[1] Alnylam Pharmaceuticals. (2023). Onpattro (patisiran) prescribing information.
[2] IQVIA. (2022). Market overview of hereditary transthyretin amyloidosis therapies.
[3] U.S. Food & Drug Administration. (2018). FDA approves Onpattro to treat a rare genetic disease.
[4] American Heart Association. (2021). Epidemiology of transthyretin amyloidosis.
[5] EvaluatePharma. (2022). RNAi therapeutics market forecast.
