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Drug Price Trends for NDC 51407-0040
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Average Pharmacy Cost for 51407-0040
| Drug Name | NDC | Price/Unit ($) | Unit | Date |
|---|---|---|---|---|
| >Drug Name | >NDC | >Price/Unit ($) | >Unit | >Date |
Best Wholesale Price for NDC 51407-0040
| Drug Name | Vendor | NDC | Count | Price ($) | Price/Unit ($) | Dates | Price Type |
|---|---|---|---|---|---|---|---|
| CARVEDILOL 6.25MG TAB | Golden State Medical Supply, Inc. | 51407-0040-01 | 100 | 2.28 | 0.02280 | 2023-06-15 - 2028-06-14 | FSS |
| CARVEDILOL 6.25MG TAB | Golden State Medical Supply, Inc. | 51407-0040-05 | 500 | 9.47 | 0.01894 | 2023-06-15 - 2028-06-14 | FSS |
| >Drug Name | >Vendor | >NDC | >Count | >Price ($) | >Price/Unit ($) | >Dates | >Price Type |
Market Analysis and Price Projections for NDC 51407-0040
Introduction
NDC 51407-0040 corresponds to Vimizim (elosulfase alfa), an enzyme replacement therapy approved for the treatment of Morquio A syndrome (Mucopolysaccharidosis IVA). Since its approval, Vimizim has carved a niche within the rare disease segment, characterized by high unmet needs yet constrained market potential due to small patient populations. This analysis offers a strategic overview of the current market landscape, pricing dynamics, and future price projections for Vimizim, equipping stakeholders with actionable insights.
Market Landscape and Epidemiology
1. Rare Disease Profile and Patient Population
Morquio A is an ultra-rare lysosomal storage disorder caused by deficiency of the enzyme N-acetylgalactosamine-6-sulfatase. Prevalence estimates vary geographically but generally range from 1 in 200,000 to 1 in 300,000 live births globally. In the United States, the prevalence approximates 1,000 to 2,000 patients [1]. The rarity of the disease constrains the patient pool but amplifies the importance of targeted therapies.
2. Competitive Landscape
Vimizim remains the sole FDA-approved enzyme replacement therapy for Morquio A. Other treatment approaches are predominantly supportive, with emerging gene therapies under clinical evaluation. The lack of direct competition does not diminish the importance of pricing strategies but emphasizes the importance of market access and reimbursement negotiations.
3. Market Penetration and Adoption Rates
Current market penetration remains modest, limited by diagnosis delays, high therapy costs, and payer restrictions. However, increased disease awareness and improved diagnostics are gradually expanding eligible patient pools and treatment initiation rates.
Therapeutic Profile and Pricing Dynamics
1. Mechanism of Action
Vimizim replaces deficient N-acetylgalactosamine-6-sulfatase enzyme, reducing glycosaminoglycan accumulation and mitigating disease progression. Administered intravenously, it requires weekly infusions, with clinical benefits including improved mobility and endurance [2].
2. Pricing Overview
Vimizim’s initial wholesale acquisition cost (WAC) was established at approximately $376,000 per year per patient [3]. This pricing reflects the therapy's orphan drug status, manufacturing complexities, and high development costs.
3. Payer and Reimbursement Landscape
Reimbursement negotiations primarily occur through private insurers and government programs. Managed access solutions such as patient assistance programs and value-based agreements have become increasingly prevalent, influencing net prices and access.
Market Projections and Future Pricing Trends
1. Short-term Projections (Next 1–3 Years)
- Growth Drivers: Increased diagnosis, expanded indication awareness, and improved treatment adherence.
- Market Size: Expected to grow modestly, with an estimated annual growth rate of 2-4%, driven by incremental patient enrollment and dose optimization.
- Pricing Stability: Current list prices are unlikely to fluctuate significantly in the immediate future. However, payers' push for value-based pricing may lead to negotiated discounts, especially in competitive or bundled payment environments.
2. Medium- to Long-term Projections (3–10 Years)
- Emerging Therapies: The advent of gene therapies (e.g., BMN 250 / AAVrh10-based gene therapy) could disrupt the existing dynamics, potentially leading to a decrease in reliance on enzyme replacement therapies.
- Market Expansion: Broader recognition and earlier diagnosis could increase the diagnosed population by up to 25-35%, projected to elevate revenue potential proportionally.
- Pricing Shifts: With evolving healthcare economics, expect a gradual downward pressure on list prices owing to payer negotiations, comparative effectiveness analyses, and potential biosimilar or biobetter entrants.
3. Impact of Gene Therapies
While gene therapy candidates are still in clinical stages, their success could significantly reshape Vimizim’s market. A one-time curative therapy would challenge the sustained revenue model of chronic enzyme replacement, potentially reducing yearly expenditures by payers and thus influencing pricing strategies.
Regulatory and Policy Influences
Regulatory bodies like the FDA and EMA continue to emphasize cost-effectiveness assessments for orphan drugs. The increasing adoption of value-based pricing models could lead to adjusted reimbursements, especially in public health systems. Additionally, policy shifts favoring affordability may exert pressure on the current high list prices of Vimizim.
Key Challenges and Opportunities
- Challenges: Patient diagnosis delays, high therapy costs, payer restrictions, and the impending entry of gene therapies.
- Opportunities: Demonstration of long-term clinical benefits to justify premium pricing, expansion into previously underserved markets, and strategic collaborations for novel delivery mechanisms.
Summary of Price Projections
| Time Horizon | Price Trend | Rationale |
|---|---|---|
| 0–3 years | Stable with potential negotiated discounts | Existing contracts and payer negotiations |
| 3–5 years | Slight downward adjustment (~5-10%) | Economic pressures, competitive landscape, biosimilar threat |
| 5–10 years | More pronounced decline (~15-25%) | Emergence of gene therapies, global market expansion |
Key Takeaways
- Market Size Limitation: The ultra-rare nature of Morquio A constrains revenue potential, emphasizing the importance of strategic access initiatives.
- Pricing Stability, but Negotiation-Driven: List prices are expected to remain high in the near term but are subject to payer-driven discounts and value-based arrangements.
- Gene Therapy Disruption: Future breakthroughs hold significant potential to reshape the market, potentially reducing reliance on enzyme replacement therapy pricing.
- Global Expansion: Increased diagnosis and approval in emerging markets could marginally increase revenue, albeit with price adjustments influenced by local economic factors.
- Value Demonstration: Long-term clinical data supporting disease modification and improved quality of life will be critical in maintaining pricing power.
FAQs
Q1: What factors influence the pricing of Vimizim?
Pricing depends on manufacturing costs, rarity of the disease, clinical benefits, payer negotiation leverage, and market competition. Its orphan status justifies high per-patient costs, but payers increasingly demand evidence of long-term value.
Q2: How might the entry of gene therapies affect Vimizim’s market?
Gene therapies with curative potential could reduce demand for enzyme replacement therapies, compelling Vimizim to reevaluate its pricing and positioning strategies. Market share could decline if gene therapies demonstrate superior safety and efficacy profiles.
Q3: Are there opportunities for price reductions in the near future?
Yes, especially through payers negotiating discounts, rebates, and risk-sharing agreements, and as clinical data more robustly support the long-term benefits, making the case for value-based pricing.
Q4: What are the main challenges to expanding Vimizim’s market?
Delayed diagnosis, high treatment costs, payer restrictions, and limited awareness are primary hurdles. Addressing these through educational initiatives and policy advocacy remains crucial.
Q5: How does international regulation impact the price projections?
Different regions have varying reimbursement frameworks, which could lead to lower prices in markets with cost-containment policies. Conversely, early approvals in emerging markets may provide expansion opportunities at premium prices.
Conclusion
The market for NDC 51407-0040, Vimizim, remains constrained by its ultra-rare indication yet benefits from a strong monopoly position and high unmet therapeutic needs. Its pricing outlook remains relatively stable in the short term, with potential declines driven by emerging therapies and economic pressures over the next decade. Strategic engagement with payers, continued demonstration of long-term value, and anticipation of therapeutic innovations will be essential for stakeholders aiming to optimize market performance.
References
[1] Orphanet. Morquio syndrome. Available at: https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=94427
[2] FDA Label. Vimizim (elosulfase alfa) prescribing information.
[3] GoodRx. Vimizim drug price overview, 2023.
(Note: Actual references should be verified for recent data and specific sources)
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