Last updated: March 4, 2026
What is the drug associated with NDC 65145-0139?
NDC 65145-0139 corresponds to Patisiran (Onpattro), an RNA interference (RNAi) therapeutic developed by Alnylam Pharmaceuticals. It is approved for the treatment of polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis).
Market Overview
Therapeutic Area and Competition
Patisiran competes primarily with Tafamidis (Vyndaqel, Vyndamax), approved for transthyretin amyloidosis, and Inotersen (Waylivra), another RNAi drug from Ionis Pharmaceuticals. The market for hereditary transthyretin amyloidosis is estimated to be worth approximately $700 million globally in 2022.
Patient Population
- Estimated prevalence of hereditary transthyretin amyloidosis: approximately 50,000 to 100,000 patients worldwide.
- Diagnosed patients in the U.S.: roughly 6,000 to 8,000.
- Treatment-eligible patients: 70-80% of diagnosed cases.
Geographic Adoption
- U.S. accounts for approximately 60-65% of sales.
- Europe and Japan represent an additional 25-30%.
- Rest of the world has limited adoption due to access and cost constraints.
Regulatory Status and Market Penetration
- Patisiran received FDA approval in 2018.
- Approved by EMA in 2019.
- Penetration remains limited by high cost, which exceeds $450,000 per year per patient.
Price Trends
Current Pricing
- Approximate list price: $450,000 to $500,000 per year in the U.S.
- Managed care discounts reduce net prices by 20-30%.
Pricing Factors
- Specialty pharmacy agreements.
- Patient assistance programs.
- Reimbursement policies vary across regions.
Price Projections
| Year |
Estimated Average Price (USD) |
Notes |
| 2023 |
$420,000 - $480,000 |
Pricing stabilizes; market remains price inelastic |
| 2025 |
$410,000 - $470,000 |
Slight price erosion expected due to biosimilars/competitors |
| 2030 |
$380,000 - $440,000 |
Potential price reduction with increased biosimilar competition and generic access |
Market Growth Drivers
- Increased diagnosis rates.
- Broader insurance coverage.
- Expansion into new indications, such as cardiomyopathy associated with ATTR.
Revenue Projections
Short-term (2023-2025)
- Sales expected to grow at a CAGR of 4-6% driven by increased patient access.
- Estimated global sales revenue: $350 million to $500 million in 2023.
- U.S. accounts for approximately 60% of sales.
Long-term (2026-2030)
- Market penetration increases with expanded indication approvals.
- Competition from generic amino acid-based therapies could apply downward pressure.
- Projected global sales revenue by 2030: approximately $750 million to $1 billion.
Risk Factors and Market Dynamics
- Entry of biosimilars or generics may decrease prices.
- Changes in reimbursement policies could impact profitability.
- Improved diagnostics may accelerate market share growth.
- Development of new, more effective treatments could diminish market size.
Key Takeaways
- NDC 65145-0139 represents Patisiran, a leading RNAi therapy for hATTR amyloidosis.
- Current U.S. list prices hover around $450,000 annually, with flat to slight declining trends projected.
- The global market remains consolidating, with growth driven by increased diagnosis and expanded indications.
- Price erosion is anticipated as biosimilar options develop, but market growth hinges on diagnosis rates and reimbursement policies.
FAQs
1. What factors influence Patisiran’s pricing trajectory?
Pricing stability is influenced by market size, competition, reimbursement negotiations, and biosimilar entry.
2. How does Patisiran compare to its competitors?
It is one of the first approved RNAi therapies for hereditary transthyretin amyloidosis, with high efficacy and significant unmet needs. Competition includes tafamidis, which is priced slightly lower, and emerging biosimilars.
3. What is the potential impact of biosimilars?
Biosimilar products could reduce prices by 20-40% over the next 5-7 years, impacting revenue and market share.
4. What are the main markets for Patisiran?
The U.S., European Union, and Japan are primary markets, accounting for about 90% of sales.
5. How might regulatory changes affect prices?
Reimbursement reforms and pricing negotiations may lead to further price reductions, especially in Europe and Asia, where regulatory frameworks are evolving.
References
- Alnylam Pharmaceuticals. (2022). Patisiran (Onpattro) Prescribing Information.
- IQVIA. (2022). Global Pharmaceutical Market Reports.
- EvaluatePharma. (2022). HEREDITARY TRANSTHYRETIN AMYLOIDOSIS Market Forecast.
- U.S. Food & Drug Administration. (2018). FDA approval of Patisiran.
- European Medicines Agency. (2019). EMA approval of Patisiran.
