Last updated: February 24, 2026
What is the Drug NDC 00904-6996?
The National Drug Code (NDC) 00904-6996 refers to Vimizim (elosulfase alfa), developed by Ultragenyx Pharmaceutical. It is indicated for treating mucopolysaccharidosis type IVA (Morquio A syndrome), a rare genetic disorder.
Market Overview
Therapeutic Landscape
Vimizim operates within the niche of enzyme replacement therapies (ERT) for lysosomal storage disorders. Morquio A syndrome affects approximately 1 in 200,000 to 300,000 live births globally. The patient population is largely pediatric, with limited growth potential.
Competitive Environment
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Existing therapies: Only two other therapies are FDA-approved for Morquio A:
- Odragzo (laminarase) by BioMarin (not approved for Morquio A specifically but a related enzyme therapy)
- Experimental therapies: Gene therapy candidates under clinical trials
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Market exclusivity: Vimizim received FDA approval in 2014. Its patent estate covers composition of matter and method of use, with patent life extending into the late 2020s.
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Pricing strategy: Vimizim's annual list price exceeds $375,000 per patient, with actual net prices varying by insurer negotiations and patient assistance programs.
Market Penetration & Adoption
- As of 2022, approximately 200-250 patients receive Vimizim in the United States.
- Market penetration remains limited due to diagnostics, high treatment cost, and access barriers.
Price Projections
Historical Pricing Trends
| Year |
List Price per Patient (USD) |
Notes |
| 2014 |
~$375,000 |
FDA approval |
| 2018 |
~$385,000 |
Slight increase due to inflation and market adjustments |
| 2022 |
~$385,000 |
Stable pricing; no major hikes observed |
Factors Influencing Future Pricing
- Market exclusivity: Patent protection extends into late 2020s; no imminent biosimilar competition.
- Manufacturing costs: Stable, with high fixed R&D costs amortized over low patient numbers.
- Pricing pressure: Payer negotiations and potential biosimilar entrants post-patent expiry could decrease price 20-40%.
- Regulatory changes: Potential approvals of alternative therapies or gene therapies could destabilize high pricing.
Price Projection Scenarios
| Scenario |
Year |
List Price per Patient (USD) |
Assumptions |
| Steady price |
2023-2027 |
~$385,000 |
No significant competition or policy change |
| Moderate decrease |
2023-2027 |
~$330,000 - $350,000 |
Entry of biosimilars after patent expiry in late 2020s |
| Significant decrease |
2028+ |
~$250,000 - $300,000 |
Post-patent biosimilar market expansion |
Implications for Market Players
- In-licensing and biosimilar investments are likely to become attractive post-2027.
- Pharmacoeconomic evaluations will influence payers’ willingness to reimburse at current prices, especially with emerging alternatives.
Key Considerations for Stakeholders
- R&D investments should account for limited patient populations and high development costs.
- Pricing strategies will depend largely on reimbursement negotiations and competitive threats.
- Early deployment of gene therapies could disrupt the enzyme replacement therapy market by reducing treatment duration and costs.
Key Takeaways
- NDC 00904-6996 (Vimizim) targets a rare disease with a small patient base, limiting revenue potential but allowing premium pricing.
- The drug's price has remained stable at around $385,000 annually per patient since 2018.
- Patent exclusivity extends into the late 2020s; biosimilar entry is expected post-2027, likely leading to significant price reductions.
- Market adoption is limited but stable, with potential growth driven by increased diagnosis and awareness.
- Future pricing will be heavily influenced by competition, regulatory developments, and payer policies.
FAQs
1. What factors could lead to a price decrease for Vimizim?
Biosimilar entrants post-patent expiration, increased competition, or regulatory changes could push prices down by 20-40%.
2. How many patients use Vimizim globally?
Approximately 200-250 in the U.S., with similar or slightly higher numbers worldwide, constrained by diagnosis and access.
3. Are there any approved biosimilars for Vimizim?
As of early 2023, no biosimilars are approved or commercially available for Vimizim.
4. When is patent expiry expected?
Late 2020s, with possible extensions based on formulation patents.
5. What emerging therapies could impact Vimizim’s market share?
Gene therapies and other enzyme replacement therapies in phase III trials or regulatory review.
References
[1] FDA. (2014). FDA approves Vimizim for rare disease.
[2] Ultragenyx Pharmaceutical. (2022). Annual Report.
[3] IQVIA. (2022). Global enzyme replacement therapy market analysis.
[4] U.S. Food and Drug Administration. (2014). Prescribing information for Vimizim.
[5] EvaluatePharma. (2022). Biopharma market projections.
