Last updated: March 15, 2026
What is the Drug NDC 13668-0485?
NDC 13668-0485 refers to Onpattro (patisiran), an intravenous medication approved by the FDA in August 2018. It treats hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), a rare, progressive disease. Patisiran is a lipid nanoparticle siRNA therapy developed by Alnylam Pharmaceuticals.
Market Overview
Indications and Market Size
Onpattro targets a rare, genetically inherited amyloidosis affecting approximately 50,000 patients globally. In the U.S., prevalence estimates hover around 10,000 cases, but actual diagnosed numbers are lower due to limited awareness and testing.
Therapeutic Landscape
Prior to Onpattro, treatment options for hATTR amyloidosis were limited, mainly supportive care, with liver transplant being an option for some cases. Other experimental therapies, such as antisense oligonucleotides and small molecule stabilizers, compete in the space or aim to expand treatment options.
Competitive Position
- Key Competitors: Novo Nordisk's Dexpramipexole (not yet approved), Akcea's Tegsedi (inotersen), and upcoming gene-silencing agents.
- Market Entry Barriers: Orphan drug designation provides market exclusivity until 2025-2027. Price Reimbursement depends on payer negotiations and regional policies.
Current Pricing and Revenue Data
List Price
- The average wholesale price (AWP) for Onpattro is approximately $450,000 to $500,000 annually per patient, based on dosing of 0.3 mg/kg every three weeks for an average patient weight of 70 kg.
Revenue Figures
- 2019: $230 million
- 2020: $370 million
- 2021: $510 million
- 2022: $650 million (estimates based on sales reports)
Market Penetration
- U.S. sales constitute 60-70% of global revenue.
- Patient uptake remains limited, with an estimated 2,000-3,000 treated patients globally by 2022.
Price Projections and Market Growth
Short-Term Projections (2023-2025)
- Price Stability: No significant change expected due to high developmental and manufacturing costs, and existing patent protections.
- Market Expansion: Pivotal trial data for a subcutaneous formulation and next-generation RNAi therapies could impact pricing strategies. If the subcutaneous option is approved in 2024, price adjustments may occur to reflect convenience benefits.
Long-Term Outlook (2026-2030)
- Potential Price Adjustments: Entry of biosimilars or competing therapies might pressure prices downward by 10-20% over the next five years.
- Market Growth: Driven by increased diagnosis rates, formulary approvals, and expanded indications such as ATTR cardiomyopathy.
- Global Expansion: Emerging economies could see price points around 25-40% lower than U.S. levels, depending on regulatory policies and healthcare economics.
Key Factors Affecting Future Pricing
| Factor |
Impact |
Notes |
| Regulatory exclusivity |
Keeps prices high for initial years |
Patent and orphan drug status protect pricing until mid-2020s |
| Competitive drugs |
Erode market share |
New therapies may compete on efficacy, delivery, and pricing |
| Manufacturing costs |
Stabilize or increase prices |
Top-tier technology and production expenses |
| Payment and reimbursement policies |
Potential price caps |
Value-based pricing might be adopted |
Strategic Considerations
- Market Expansion: Focus on geographic regions with emerging healthcare infrastructure and high unmet need.
- Pricing Strategy: Maintain premium pricing with value-based contracts emphasizing clinical outcomes.
- Pipeline Development: Monitor for approvals of next-generation RNAi or gene-editing therapies that could disrupt current pricing.
Key Takeaways
- NDC 13668-0485 (Onpattro) generates revenues exceeding $650 million annually, with stable pricing around $450,000-$500,000 per patient.
- The market remains niche owing to disease rarity, but growth is driven by increased diagnosis, expanded indications, and new formulations.
- Price projections suggest stability through 2025, with potential decreases from biosimilars or emerging therapies starting around 2026.
- Competitive pressures, regional price negotiations, and healthcare policies will influence long-term pricing trends.
- Broad global adoption depends on economic factors and regulatory environments, especially outside the U.S.
FAQs
1. Will the price of Onpattro decrease in the coming years?
Likely, starting around 2026, due to possible biosimilar entry and market competition, with a potential 10-20% reduction over five years.
2. What factors could lead to an increase in Onpattro’s price?
Limited competition, high manufacturing costs, and expanding indications without new competitors could sustain or increase prices temporarily.
3. How is the treatment uptake expected to change?
Treatment uptake is projected to increase with improved diagnosis methods and expanded treatment indications, potentially doubling patient numbers over the next five years.
4. Are there upcoming therapies that could replace Onpattro?
Next-generation RNAi therapies, gene editing approaches, and drugs like Akcea’s inotersen may compete or replace Onpattro depending on efficacy, safety, and cost.
5. How do regional policies affect pricing?
Regions with strict price controls, such as parts of Europe, may see significantly lower prices compared to the U.S. market, where payers emphasize value-based agreements.
References
[1] Alnylam Pharmaceuticals. (2023). Onpattro (patisiran) prescribing information.
[2] IQVIA. (2022). Global sales reports.
[3] FDA. (2018). Approval of Onpattro for hereditary transthyretin-mediated amyloidosis.
[4] EvaluatePharma. (2022). Pharmaceutical market forecast reports.
