Last updated: February 16, 2026
Overview of NDC 50268-0050
NDC 50268-0050 is the product code for Vimizim (elosulfase alfa), a recombinant human enzyme used to treat Morquio A syndrome (mucopolysaccharidosis IV A). Approved by the FDA in 2014, it is marketed by Ultragenyx Pharmaceutical and registered with multiple regulatory agencies globally.
Market Size and Epidemiology
Morquio A Syndrome Incidence:
- Occurs at approximately 1 in 200,000 to 300,000 live births globally.
- United States estimates suggest between 200-400 affected individuals.
Patient Population:
- Estimated U.S. prevalence: approximately 300 patients.
- Underdiagnosis remains an issue; actual numbers could be higher.
Market Potential:
- Based on the U.S. market, potential revenue hinges on diagnosis rate, pricing, payer coverage, and therapy adherence.
- Global prevalence estimates indicate a larger but less penetrated market, mainly in Europe, Asia, and Latin America.
Price Setting and Revenue Models
Current U.S. Wholesale Acquisition Cost (WAC):
- Approximate annual list price is $375,000 to $400,000 per patient (sources include SSR Health and Magellan).
- Price margins vary based on discounts, rebates, and payer negotiations.
Pricing Trend Analysis:
- No substantial increase in price since approval, reflects rarity-driven pricing models.
- Similar enzyme replacement therapies (ERTs) like Elaprase (idursulfase) and Naglazyme (galsulfase) have maintained stable pricing over recent years.
Market Access and Reimbursement:
- Payer coverage includes both private insurers and Medicaid, with prior authorization often required.
- Rare disease programs often benefit from expanded access and special funding programs.
Competition and Market Share
Primary Competitors:
- No direct enzyme replacement competitors for Morquio A.
- Several orphan drugs with similar rarity profiles exist but target different conditions.
Pipeline and Future Competition:
- No approved gene therapy or other ERTs for Morquio A as of 2023.
- Limited pipeline suggests Vimizim will retain market dominance in the near term.
Price Projections (2023–2028)
| Year |
Estimated Average Price (per patient) |
Key Factors |
| 2023 |
$375,000 – $400,000 |
Stable pricing, high payer barriers |
| 2024 |
$375,000 – $410,000 |
Slight upward pressure due to inflation, reimbursement trends |
| 2025 |
$380,000 – $420,000 |
Potential for negotiated discounts, market expansion efforts |
| 2026 |
$385,000 – $430,000 |
Emerging payer negotiations, inflation considerations |
| 2027 |
$390,000 – $440,000 |
Increased awareness, broader diagnosis rates |
| 2028 |
$395,000 – $450,000 |
Continued inflation adjustment, new payer policies |
Key Assumptions:
- No significant price reductions or biosimilar entry.
- Steady diagnosis rates, with incremental market expansion.
- Reimbursement policies remain favorable to rare disease treatments.
Market Drivers & Barriers
Drivers:
- Increasing awareness leading to improved diagnosis.
- Growing global rare disease infrastructure.
- Limited competition preserves market share.
Barriers:
- High treatment cost impacting healthcare budgets.
- Payer resistance to renew access without substantial evidence.
- Diagnostic delays limiting patient access.
Strategic Considerations
- Potential for value-based pricing hinges on long-term outcomes data.
- Expansion into emerging markets through localized reimbursement efforts.
- Monitoring regulatory pathways for gene therapies or alternative treatments.
Summary
NDC 50268-0050, Vimizim, holds a stable position within the rare disease treatment landscape. The annual treatment price remains around $375,000–$400,000, with a likelihood of gradual annual increases reflecting inflation and market dynamics. The overall market size remains constrained by the disease's rarity, but the unmet need and lack of competitors sustain revenue prospects.
Key Takeaways
- The rare disease status confines the market to a few hundred patients in the U.S., with global potential slightly larger.
- Current pricing remains stable, with slow incremental increases projected over the next five years.
- Market growth depends on better diagnosis rates, expanded access, and potential pipeline advancements.
- No biosimilar competition exists yet; Vimizim has an established monopoly in Morquio A.
- Reimbursement challenges persist; favorable payer negotiations are crucial for revenue stability.
FAQs
1. What factors could lead to significant price reductions for Vimizim?
Entry of biosimilars, regulatory changes, or economic pressures could compel price reductions. However, current indications and lack of competitors reduce this risk.
2. How does the global prevalence impact the revenue projections?
Limited prevalence restricts total addressable market size. Expansion in emerging markets and improved diagnosis could incrementally increase revenues.
3. Are there any approved gene therapies for Morquio A?
As of 2023, no gene therapies for Morquio A are approved. Several pipeline candidates are in early phases but face regulatory and technical hurdles.
4. How do payers view high-cost orphan drugs like Vimizim?
Payers often require prior authorization, demonstrate clinical benefit, and negotiate discounts. Reimbursement is contingent on demonstrating value relative to cost.
5. What is the outlook for pipeline competitors?
The pipeline for Morquio A remains limited, with no major advances expected soon. The market is expected to sustain Vimizim's dominance.
References
- FDA. Vimizim (elosulfase alfa) prescribing information. 2014.
- SSR Health. Estimated wholesale acquisition costs (WAC) for rare disease drugs. 2023.
- Magellan Rx Management. Pricing and reimbursement insights for enzyme replacement therapies. 2023.
- Orphanet. Morquio syndrome (Mucopolysaccharidosis IV). 2022.
- Ultragenyx Pharmaceuticals. Vimizim investor presentation. 2023.
