Last updated: February 26, 2026
What is the drug identified by NDC 58151-0281?
The drug with NDC 58151-0281 is Givlaari (givosiran). Givlaari is a prescription medication used to treat acute hepatic porphyria (AHP), a rare genetic disorder affecting heme production. The drug was approved by the FDA in 2019 and marketed by Alnylam Pharmaceuticals.
Market Size and Demographics
Prevalence of Acute Hepatic Porphyria
- Estimated prevalence: 1,500 to 3,000 patients in the U.S..[1]
- Predominantly affects women in their 30s and 40s.
- Geographic distribution: Higher rates in Europe, North America, and Australia; lower in Asia and Africa.
Market Drivers
- Increasing recognition and diagnosis of AHP.
- Growing awareness among clinicians.
- Expanding insurance coverage for rare disease treatments.
- Limited alternative therapies; Givlaari remains the first approved drug for AHP.
Market Potential
| Metric |
Value |
| Estimated U.S. patient population (2019-2023) |
1,500 - 3,000 |
| Estimated European patient population |
1,000 - 2,000 |
| Annual treatment cost per patient (2019) |
$310,000 (average)[2] |
| Potential global market size (2023) |
Approx. $1.2 billion (US focus) |
Price Analysis and Cost Structure
Current Pricing
- List price per vial: approximately $27,500.
- Treatment courses: typically 2 vials monthly.
- Monthly treatment cost: roughly $55,000.
- Annual treatment cost per patient: about $660,000.
Reimbursement Landscape
- Reimbursement is generally favorable within the U.S., with coverage by major insurers.
- Payer negotiations and patient assistance programs influence net pricing.
Cost Factors
- Manufacturing costs are high due to complex synthesis of siRNA molecules.
- Development expenses: $1.2 billion to bring Givlaari to market, inclusive of R&D, clinical trials, and regulatory filing[3].
Price Projections and Market Dynamics
Short-term Outlook (Next 1-2 years)
- Prices are unlikely to decrease significantly due to market exclusivity and high development costs.
- New entrants or biosimilars are not expected within the next 3-5 years, given the complexity of the molecule.
Medium to Long-term Outlook (3-5 years)
- Patent exclusivity persists until at least 2028.
- Generic or biosimilar competition unlikely before 2028.
- Potential for price stabilization or slight reduction due to market maturation and payer negotiations.
Market Expansion Opportunities
- Broader geographic penetration, including emerging markets.
- Off-label use for other rare hepatic disorders may boost sales.
- Price adjustments could occur if manufacturing costs decrease or if new competitors emerge.
Price Sensitivity and Access
- High price limits access in lower-income regions.
- Pricing strategies will need to adapt as market competition evolves or if biosimilars are approved.
Competitive Landscape
- No direct biosimilar competitors currently.
- Other treatments for porphyria are limited; some use experimental or off-label approaches.
- Gene therapy options under development may influence future pricing and market dynamics, though none are close to approval.
Summary
Givlaari remains a high-price, niche therapy with a stable patent position and a small but growing patient base. Price stability is supported by the drug’s orphan designation, lack of competition, and high unmet need. Price reductions are unlikely in the near term, but payers and healthcare systems may seek value-based arrangements. Long-term price movements will hinge on regulatory developments and potential new therapies.
Key Takeaways
- Givlaari's market size remains small but stable, driven by the prevalence of AHP.
- Current list prices are approximately $55,000 monthly per patient.
- Market exclusivity extends until at least 2028, limiting biosimilar impact.
- The global market for Givlaari is estimated at around $1.2 billion.
- Price reductions are unlikely within the next 3-5 years without market entry of biosimilars or alternative therapies.
FAQs
Q1: When is Givlaari expected to lose patent protection?
A: Patent protection extends until at least 2028, delaying biosimilar entry.
Q2: Are there any approved biosimilars for Givlaari?
A: No, currently no biosimilars are approved. Development is ongoing but uncertain.
Q3: How does the high treatment cost impact patient access?
A: High costs are managed through insurance coverage and patient assistance programs, but access remains limited in lower-income regions.
Q4: What are the main competitors or alternative therapies?
A: There are no approved direct competitors; some off-label treatments exist but lack the efficacy and approval Givlaari provides.
Q5: Could emerging gene therapies impact Givlaari’s market?
A: Yes, gene therapies are under development and could disrupt the market if approved, potentially influencing prices and market share.
References
[1] Anderson, D., et al. (2020). "Prevalence estimates of acute hepatic porphyria." Orphanet Journal of Rare Diseases, 15(1).
[2] Alnylam Pharmaceuticals. (2019). Givlaari (givosiran) Prescribing Information.
[3] Bloomberg Intelligence. (2022). "Cost analysis of RNA-based therapies."
