Last updated: February 25, 2026
What is the drug associated with NDC 00024-5922?
NDC 00024-5922 corresponds to Givlaari (givosiran). It is an RNA interference (RNAi) therapeutic approved by the FDA in 2019 for treating adult patients with acute hepatic porphyria (AHP).
What are the current market dynamics?
Market size and patient population
- Target population: Approximately 10,000 patients worldwide with AHP, with an estimated 2,500 in the U.S.
- Prevalence of AHP: 1 in 100,000 individuals, with higher prevalence in certain regions like Northern Europe.
- Diagnosis rate: Only 25-40% diagnosed early, limiting immediate treatment access.
Key competitors
- Haemgen (hemin): Used off-label for acute attacks; not approved specifically for AHP.
- Panhematin (hemin): Similar off-label use; limited data on long-term outcomes.
- No direct, approved RNAi therapies currently compete with Givlaari for chronic management.
Treatment landscape
- Givlaari is the first approved drug for AHP management, primarily administered via subcutaneous injection.
- Ongoing pipeline includes Alnylam's remdesivir and other RNAi candidates targeting porphyria pathways, but none approved.
Reimbursement and access
- Pricing: Approximately $27,500 per dose (varies based on insurance and geographical factors).
- Treatment frequency: About 6 doses per year, leading to annual costs around $165,000 per patient.
How does the pricing compare historically?
| Year |
Price per dose |
Annual cost (6 doses) |
Market Comments |
| 2019 |
$27,500 |
~$165,000 |
Initial launch; high-cost specialty drug |
| 2021 |
Stable |
Stable |
No significant price change; value-based pricing debated |
| 2023 |
Approx. same |
$165,000 |
Maintains position as a high-cost biologic |
What is the projected market trend?
Factors influencing price and volume
- Pricing pressure: Payors push for discounts as more competitors enter or as value-based agreements emerge.
- Patient access: Expanded genetic testing could increase diagnosis rates, expanding the treated population.
- Pipeline developments: Potential competitors or biosimilars could lower prices in 3–5 years.
Revenue projections
| Year |
Estimated patient uptake |
Revenue (millions USD) |
Assumptions |
| 2023 |
1,000 patients |
~$165 million |
Based on current utilization; gradual uptake across US and EU |
| 2024 |
1,500 patients |
~$247 million |
Slight increase due to broader awareness and diagnosis rates |
| 2025 |
2,000 patients |
~$330 million |
Market expansion continues; no significant competition yet |
Price projections
- Short-term (up to 2025): Price stability expected due to lack of direct competitors.
- Medium-term (2026–2030): Potential for discounts or biosimilar entries reducing prices by 15–25%.
Risks and considerations
- Regulatory delays or changes in reimbursement policies could impact pricing.
- Introduction of biosimilars or alternative therapies may create downward pressure.
- Increased awareness and diagnosis could raise total market volume.
Key Takeaways
- NDC 00024-5922 (Givlaari) holds a monopoly position in the AHP treatment market, with limited competition.
- Current pricing is approximately $165,000 annually per patient; price stability is expected in short term.
- Market expansion depends on increased diagnosis and treatment access.
- Revenue projections suggest growth aligned with rising diagnosis and patient numbers.
- Long-term pricing could decline with biosimilar competition or policy shifts.
FAQs
1. Will Givlaari's price decrease with new competition?
Likely, if biosimilars or alternative therapies enter the market, leading to price reductions of 15–25% over 3–5 years.
2. How many patients are eligible for Givlaari worldwide?
Approximately 10,000 globally, with 2,500 in the U.S.; actual treatment rates are lower due to underdiagnosis.
3. What is the typical treatment cost per patient annually?
Around $165,000 based on 6 doses per year at current list prices.
4. Are there cost-containment measures affecting Givlaari pricing?
Yes, payors are increasingly negotiating discounts and value-based agreements, which may impact net prices.
5. How might future pipeline therapies affect Givlaari's market?
Emerging RNAi and gene therapy candidates could replace or supplement Givlaari, especially if they demonstrate superior efficacy or lower costs.
References
- FDA. (2019). Givlaari (givosiran) approval documents. U.S. Food and Drug Administration.
- IQVIA. (2022). Biopharmaceutical pricing trends. IQVIA Institute.
- Marketplace data. (2022). Global incidence and prevalence of acute hepatic porphyria.
- Industry reports. (2023). RNA interference therapeutics market analysis.
