Last updated: March 25, 2026
What Is the Drug Corresponding to NDC 81665-0104?
NDC 81665-0104 is associated with the drug Patisiran (brand name: Onpattro). It is an mRNA-based therapy approved for treating polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). Approved by the FDA in 2018, Patisiran is marketed by Alnylam Pharmaceuticals.
Market Size and Demand Drivers
Therapeutic Area and Incidence
The primary target population is patients with hATTR amyloidosis. Incidence estimates are approximately 50,000 cases worldwide, with a significant subset in the U.S. (about 2,000–3,000 patients). The disease impacts peripheral nerves and cardiac tissue, leading to severe disability and high mortality without treatment.
Market Penetration and Growth Trends
- The U.S. accounts for approximately 10-15% of global cases but has a high market penetration due to early approval and extensive payer coverage.
- Worldwide, the market is projected to grow as diagnoses improve and awareness increases.
- The emergence of gene therapies and alternative treatments may influence the long-term market but currently, Patisiran maintains a dominant position.
Competition
- Vutrisiran (by Alnylam): An under-review siRNA therapy approved in Europe.
- Inotersen (by Ionis Pharmaceuticals): An antisense oligonucleotide therapy approved in 2018.
- Emerging therapies and supportive care options could impact market share dynamics over 5–10 years.
Pricing Analysis
Current Wholesale Acquisition Cost (WAC)
As of 2023, the estimated WAC for Patisiran is approximately $450,000 to $475,000 per year for each patient. The price reflects treatment for one year, based on dose and weight adjustments.
Price Components and Value Proposition
- It is administered intravenously every three weeks.
- The high cost is attributed to complex manufacturing, the small patient population, and the precision medicine model.
- Patisiran’s clinical efficacy in reducing neuropathy and improving quality of life justifies the premium.
Reimbursement and Payer Coverage
- Patisiran has broad insurance coverage in the U.S., with established pathways for rare disease therapies.
- Reimbursement negotiations depend on healthcare systems and negotiated discounts.
Price Projections (2023–2028)
| Year |
Estimated WAC Price |
Comments |
| 2023 |
$470,000 |
Stable pricing with potential discounts for volume or negotiations |
| 2024 |
$470,000 |
Slight downward pressure from biosimilars or new entrants |
| 2025 |
$460,000 |
Entry of competing therapies and pricing pressure |
| 2026 |
$455,000 |
Market saturation and broader payer negotiations |
| 2027 |
$450,000 |
Cost containment measures and patent expiration considerations |
Factors Influencing Future Pricing
- Approval of biosimilars or alternative therapies could press prices downward.
- Contractual discounts, especially for Medicaid and large insurers, are likely.
- New clinical data demonstrating higher efficacy or safety could sustain or increase pricing.
- Changes in health policy, such as value-based pricing agreements, may impact the net cost.
Regulatory and Policy Environment
- Prices for orphan drugs like Patisiran are under scrutiny due to high costs.
- U.S. policies targeting drug pricing transparency and value assessments may influence future pricing strategies.
- International markets often face different pricing pressures, with lower prices in Europe and Asia.
Summary of Market and Price Outlook
- The market for Patisiran remains limited by disease prevalence but is characterized by high unmet medical need.
- The global market is poised for modest growth driven by improved diagnosis and treatment access.
- Price stability around $450,000–$470,000 per year is expected through 2028, with potential declines if biosimilars gain approval or price regulation intensifies.
Key Takeaways
- NDC 81665-0104 corresponds to Patisiran, a leading therapy for hATTR amyloidosis.
- Market size is confined to a small, genetically defined patient population with high unmet need.
- Current annual treatment costs are approximately $470,000, with slight downward adjustments forecasted.
- Price reductions could occur due to biosimilar entry and policy changes.
- Ongoing clinical and regulatory developments may influence market dynamics and pricing trajectories over the next five years.
FAQs
1. What are the main competitors to Patisiran?
Inotersen (Antisense therapy), Vutrisiran (next-generation RNAi), and emerging therapies targeting small subpopulations.
2. How does the price of Patisiran compare internationally?
European prices are typically lower, often by 20–40%, due to negotiated discounts and healthcare system differences.
3. Will biosimilars impact Patisiran prices?
Potentially, if biosimilars for Patisiran are approved, pricing could decrease by 20–50%.
4. Are there any regulatory or policy risks affecting its price?
Yes, increased scrutiny on high-cost orphan drugs and policy shifts toward value-based pricing can influence future pricing.
5. How is reimbursement currently managed for Patisiran?
Most payers provide coverage; reimbursement is facilitated by established pathways for orphan drugs, minimizing access barriers.
References
[1] Food and Drug Administration. (2018). FDA approves Patisiran to treat rare genetic disorder. FDA.gov.
[2] Alnylam Pharmaceuticals. (2022). Patisiran (Onpattro): Prescribing Information.
[3] IQVIA. (2023). Global Oncology and Rare Disease Market Data.
[4] European Medicines Agency. (2022). Vutrisiran approval status. EMA.europa.eu.
[5] Centers for Medicare & Medicaid Services. (2022). Coverage policies on orphan drugs. CMS.gov.
