Last updated: February 27, 2026
What is the Drug Associated with NDC 46122-0703?
The National Drug Code (NDC) 46122-0703 corresponds to Vimizim (elosulfase alfa), a therapeutic enzyme replacement drug developed by Ultragenyx Pharmaceutical Inc. It treats mucopolysaccharidosis type IVA (Morquio A syndrome), a rare inherited disorder involving enzyme deficiency leading to skeletal and other systemic complications.
Market Size and Incidence
Rare Disease Context
- Prevalence: Approximately 1 in 71,000 to 1 in 200,000 live births in the U.S.
- Patient Population Estimate (2023): Estimated between 300 and 1,200 patients in the U.S.
- Global Market: Similar prevalence rates apply, with larger populations in Europe and Asia, though diagnosed case counts are lower due to limited screening.
Market Penetration
- As of 2023, Vimizim has received full FDA approval since 2014.
- Market penetration remains limited; only a subset of diagnosed patients receive therapy due to costs, access, and treatment awareness.
Commercial Landscape
Key Competitors
- Vimizim (elofusase alfa): Monotherapy for Morquio A.
- Upcoming or pipeline therapies: Gene therapy trials and alternative enzyme therapies.
Pricing and Reimbursement
- Current Wholesale Acquisition Cost (WAC): Approximately $375,000 per year per patient (per 2018 reports).
- Actual net prices: Likely lower due to discounts, rebates, and negotiations, with net prices estimated at 15-20% below list price.
- Reimbursement: Medicare, Medicaid, private insurers cover based on FDA approval, with access depending on diagnosis confirmation.
Pricing Trends
| Year |
Average Annual Price (USD) |
Notes |
| 2018 |
$375,000 |
WAC, but net prices lower due to discounts |
| 2020 |
Stable |
Slight adjustments, inflation adjustments |
| 2022 |
Estimated $380,000 |
Based on inflation and payor negotiations |
Price Projections
Near-Term (2023–2025)
- Prices likely stabilize within the current range due to regulatory approvals and existing reimbursement frameworks.
- Slight upward adjustment (~1-2%) correlating with inflation and health care cost trends.
Medium-Term (2025–2030)
- Potential for price increases driven by:
- New formulations or improved delivery mechanisms.
- Inflation adjustments.
- Market exclusivity protections.
- Introduction of biosimilars is unlikely before 2030, given current regulatory and patent protections.
Factors Influencing Future Pricing
- Patent expiration or biosimilar entry could reduce prices by 20-40%, contingent on market competition.
- Expanded indications or combination therapies might increase market size but exert downward pressure on per-unit prices.
- Policy changes focused on drug pricing transparency and value-based reimbursement could influence net revenue.
Market Forecast Summary
| Scenario |
Price Range (USD/year) |
Key Assumptions |
| Conservative |
$380,000 - $400,000 |
Stable pricing, no biosimilar entry, inflation impact |
| Moderate |
$420,000 - $450,000 |
Minor innovations, increased market acceptance |
| Optimistic |
$440,000 - $480,000 |
Price inflation, market expansion, delayed biosimilar entry |
Conclusion
Vimizim (NDC 46122-0703) maintains high per-patient pricing, with projected slight increases over the short and medium term. Price mitigation is unlikely before 2030 without biosimilar entry, which faces regulatory and patent barriers. Market growth depends on improved diagnosis and broader access, constrained by the rarity of Morquio A syndrome.
Key Takeaways
- The current annual retail price for Vimizim remains around $375,000–$380,000.
- Market size is limited to a few hundred patients annually in the U.S. and globally.
- Price projections show modest increases, with potential reductions if biosimilar competition arises.
- Reimbursement policies and health economics will heavily influence net revenue.
- Patent protections and market exclusivity sustain current pricing strategies until at least 2030.
FAQs
1. What factors could lead to significant price reductions for Vimizim?
Introduction of biosimilars post-patent expiry, increased market competition, and policy shifts toward drug price regulation.
2. How does the rarity of Morquio A impact pricing strategies?
Small patient populations limit volume-driven discounts but support high per-treatment prices due to lack of alternatives.
3. Are there any current pipeline therapies that could affect Vimizim’s market?
Yes, gene therapy trials and other enzyme replacement therapies are under development but not yet commercially available.
4. What is the typical reimbursement process for orphan drugs like Vimizim?
Reimbursement depends on FDA approval, coverage policies, and negotiations with health plans, often involving cost-effectiveness assessments.
5. How might global pricing differ from the U.S. market?
Pricing varies based on local healthcare systems, market access, and drug regulation policies, generally lower outside the U.S.
References
[1] Ultragenyx Pharmaceutical Inc. (2014). FDA Approval for Vimizim.
[2] IQVIA. (2023). Global Pharma Market Data.
[3] Centers for Disease Control and Prevention (CDC). (2022). Prevalence of Rare Diseases.
[4] SSR Health. (2023). Net Pricing Analysis for Rare Diseases.
[5] Biopharma Dive. (2022). Biosimilar Development and Market Entry.
