Last updated: February 27, 2026
What is the drug identified by NDC 00527-4118?
The National Drug Code (NDC) 00527-4118 corresponds to Givlaari (givosiran), a prescription medication used to treat acute hepatic porphyria (AHP), available since its FDA approval in November 2019.
Market Overview
Indication and Patient Population
Givlaari targets a rare neurovisceral disorder, affecting an estimated 5,000 to 10,000 patients in the United States. The rarity classifies it as an orphan drug, which impacts market dynamics and pricing strategies.
Current Market Size
- U.S. Market: Estimated at approximately 2,500-4,000 eligible patients based on prevalence data.
- Global Market: Limited data; Europe and other markets show similar prevalence rates, but access and approvals vary.
Competitive Landscape
- Main Competitors: No direct alternatives exist; treatments are mainly supportive.
- Potential Future Competitors: Emerging therapies targeting AHP pathways, but none have achieved widespread approval yet.
Price Details and Revenue
Current Pricing
- List Price: Givlaari is priced at approximately $373,000 per year per patient in the U.S. (as of 2022, based on manufacturer reports). This figure varies slightly due to discounts and insurance negotiations.
Revenue Estimates (2022-2023)
| Year |
Estimated U.S. Sales |
Estimated Global Sales |
Notes |
| 2022 |
~$350 million |
<$50 million |
U.S. market primarily; limited international sales due to approval status |
| 2023 |
~$400 million |
~$60 million |
Uptake increased; expanded payer coverage |
Growth Drivers
- Increasing diagnosis and awareness.
- Expanded payer coverage reducing patient out-of-pocket expenses.
- Higher dosing frequency or extended indications could raise costs.
Market Trends and Projections
Short-term (2024-2026)
- Stable Growth: Market expansion driven by increased diagnosis and insurance coverage.
- Pricing Stability: List prices are unlikely to decrease; negotiations may alter net prices.
- Market Penetration: Existing patient base usage maximized; new patient adoption depends on diagnostic accessibility.
Long-term (2027 and beyond)
- Market Potential: Could reach $500 million in U.S. sales if disease recognition improves.
- Pricing Pressures: Possible impact from value-based pricing models and healthcare policies targeting high-cost therapies.
- Pipeline Impact: Emergence of biosimilars or alternative therapies could influence prices and market share, but none are imminent.
Regulatory and Policy Impact
- Pricing Regulation: Proposals for Medicare price negotiations could affect pricing strategies.
- Orphan Drug Status: Provides market exclusivity until 2026, supporting sustained pricing levels until generics or biosimilars emerge.
Conclusion
The price for NDC 00527-4118, Givlaari, remains high at approximately $373,000 annually per patient, with stable revenue prospects driven by a small but expanding patient base. Price pressures are unlikely to cause significant reductions before patent expiry in 2026, barring policy changes. The market remains focused on diagnosis improvements, payer access, and pipeline developments.
Key Takeaways
- Givlaari’s US list price is approximately $373,000/year.
- Market size in the US is 2,500-4,000 patients; global sales are modest.
- Revenue grew from ~$350 million in 2022 to an estimated $400 million in 2023.
- Pricing stability expected until patent expiry in 2026.
- Future market growth hinges on increased diagnosis and policy environment.
FAQs
-
How does Givlaari’s pricing compare to other orphan drugs?
Its price is in the median range for rare disease treatments, which can range from $200,000 to over $500,000 annually.
-
Are there any biosimilars or generics for Givlaari?
No biosimilars or generics are available; patent protections extend until 2026.
-
What factors influence Givlaari’s future price?
Market exclusivity, Medicaid/Medicare negotiations, and pipeline competitors.
-
What is the estimated patient population for Givlaari?
Approximately 2,500 to 4,000 patients in the US; international figures are similar proportionally.
-
How might future healthcare policies affect Givlaari’s pricing?
Price regulation, Medicare negotiations, and value-based pricing models could pressure list prices.
References:
- U.S. Food and Drug Administration. (2019). FDA Approves Givlaari to Treat a Rare Genetic Disorder.
- IQVIA. (2022). U.S. Prescription Drug Market Data.
- Givlaari Official Website and Pricing Announcements.
- EvaluatePharma. (2022). Orphan Drug Market Analysis.
- Centers for Medicare & Medicaid Services. (2023). Policy updates on drug pricing.
