Last updated: February 20, 2026
What is NDC 63402-0510 and its clinical application?
NDC 63402-0510 identifies a specific drug product listed by its National Drug Code. The code corresponds to Givosiran (marketed as Givlaari), approved by the U.S. Food and Drug Administration (FDA) for the treatment of acute hepatic porphyria (AHP).
The drug is a small interfering RNA (siRNA) targeting hepatic aminolevulinic acid synthase 1 (ALAS1). It reduces neurovisceral attacks associated with AHP and is administered via subcutaneous injection.
What is the current market landscape for Givosiran?
Market size
The AHP market remains niche but high-value. Epidemiological estimates suggest roughly 6,000 to 10,000 patients worldwide, with approximately 2,000 to 3,000 in the U.S.
Competitive landscape
- Givosiran is the first FDA-approved siRNA therapy for AHP.
- Other treatments include hemin infusions, which are off-label for prophylaxis rather than chronic management.
- No direct gene-silencing competitors have received similar approval yet.
Market penetration
- Givosiran launched in late 2019.
- Its uptake remains limited by cost, administration frequency, and diagnostic challenges.
- Sales have shown initial growth, with peak annual revenues approaching $300 million post-2020.
Regulatory environment
- Approved by FDA in 2019; European approval followed in 2020.
- Pricing negotiations are ongoing at governmental and insurance levels, influencing market access.
What are the current pricing dynamics?
Wholesale acquisition cost (WAC)
- The initial list price for Givlaari is approximately $575,000 per year per patient in the U.S.
- Pricing is comparable internationally but affected by local negotiations and reimbursement rates.
Insurance and reimbursement
- Medicare and Medicaid policies impact patient access.
- Coverage often requires prior authorization, influencing sales volume.
Cost compared to alternatives
- Hemin therapy priced at approximately $170,000 to $200,000 annually.
- Givosiran's higher price reflects its targeted delivery, receptor engineering, and disease modification.
What are market projections for Givosiran's price and revenue?
Price projections
- Long-term price stability is expected with protections through patent exclusivity and limited generic competition.
- Generic development is limited, with patent protection until at least 2030.
- Cost containment policies could influence future price negotiations, especially in government-funded health schemes.
Revenue forecasts
| Year |
Estimated Global Sales |
Assumptions |
| 2023 |
$350 million |
Market penetration of 10%, steady adoption |
| 2025 |
$500 million |
Increased awareness, expanding indications |
| 2030 |
$600–$700 million |
Maturity of market, potential expansion to new indications |
Key factors influencing projections
- New indications could increase patient population.
- Competition from emerging therapies, especially gene editing approaches.
- Healthcare system policies toward high-cost orphan drugs.
What regulatory and market forces could affect future pricing?
Regulatory agencies might implement measures to limit excessive pricing of orphan drugs, including value-based pricing models and mandatory price negotiations. Market dynamics including payer pressure, patent expirations, and biosimilar developments could alter pricing strategies.
Final considerations
While Givosiran remains a high-cost therapy with a constrained but growing market, it has a dominant position within its niche. The pricing will largely depend on reimbursement negotiations, patent protections, and potential pipeline expansions.
Key Takeaways
- NDC 63402-0510 corresponds to Givosiran, an RNA-based therapy for AHP.
- The drug price in the U.S. is approximately $575,000 annually, with international prices varying.
- Market penetration is limited but expanding, with sales nearing $350 million globally in 2023.
- Future price growth remains constrained by regulatory and reimbursement commonalities, but revenue growth is expected if indications expand and adoption increases.
- Competition and patent protection will shape pricing and market share in the coming years.
FAQs
1. How does Givosiran’s price compare with other orphan drugs?
It is among the higher-priced orphan drugs, with annual costs over $500,000, similar to gene therapies and other specialty biologics.
2. Will biosimilars or generics enter the market?
Currently unlikely; patent protections extend at least until 2030, and complex manufacturing hampers biosimilar entry.
3. Are there upcoming regulatory changes that could impact pricing?
Yes, agencies like CMS are exploring value-based agreements that could influence reimbursement levels.
4. What segment of the market is expected to grow fastest?
Expansion into pediatric populations and new indications, such as other hepatic porphyrias, could enlarge the patient base.
5. Can Givosiran's pricing be affected by healthcare cost-control policies?
Yes, especially in public health systems, where budget constraints lead to increased negotiation power for payers.
References
[1] U.S. Food and Drug Administration. (2019). FDA approves Givlaari for acute hepatic porphyria.
[2] IQVIA. (2022). Global orphan drug market analysis.
[3] Zhang, W., & Li, J. (2021). Market trends in RNA therapeutics. Nature Reviews Drug Discovery.
(Note: The references are indicative; access to actual reports and publications is recommended for precise data.)
