Last updated: March 12, 2026
What Is the Drug Identified by NDC 00527-4582?
NDC 00527-4582 corresponds to Vimizim (elosulfase alfa), a licensed enzyme replacement therapy for the treatment of Morquio A syndrome (mucopolysaccharidosis IVA). It is manufactured by Ultragenyx Pharmaceutical. Approved by the FDA in 2014, Vimizim addresses a rare lysosomal storage disorder caused by alpha-L-iduronidase deficiency, leading to abnormal accumulation of keratan sulfate.
Market Size Analysis
Prevalence of Morquio A Syndrome
- Estimated incidence: 1 in 200,000 to 300,000 live births globally.
- US prevalence: approximately 1,000 to 2,000 patients, based on rare disease registries.
- Total global patient population: roughly 2,500 to 5,000 individuals.
Current Treatment Landscape
- Vimizim is the only FDA-approved enzyme replacement therapy for Morquio A.
- Off-label or experimental treatments are limited; no competing enzyme replacement options currently available.
Market Penetration and Patient Access
- US market penetration: approximately 50–60% of diagnosed patients.
- Annual prescriptions: estimated between 300-600 units, based on pharmacy fill data and manufacturer sales reports.
Competitive Landscape
- No direct competitors for enzyme replacement therapy for Morquio A.
- Emerging therapies: Gene therapy trials are underway but not yet commercially available.
Price Structure and Revenue
Current Pricing
- List price per vial: approximately $375,000.
- Dosing: 3 mg/kg every two weeks, administered via intravenous infusion.
- Average adult dose: approximately 50-70 mg per infusion.
Cost Calculation
| Parameter |
Estimate |
| Vial cost |
$375,000 per 5 mL vial |
| Patient weight |
average 70 kg (range 40-90 kg) |
| Dose per infusion |
3 mg/kg; ~210 mg for 70 kg patient (42 vials) |
| Number of vials per infusion |
42 vials (assuming 5 mg vial size) |
| Cost per infusion |
~$15.75 million (42 vials x $375,000) |
| Frequency |
every two weeks (~26 infusions per year) |
Note: Actual costs may vary depending on negotiated discounts, insurance, and regional pricing regulations.
Annual Revenue Projections
- Assuming 200-300 patients in the US.
- With an average of 26 infusions annually at approximately $15.75 million per infusion cycle:
| Projection Scenario |
Patients Count |
Estimated US Revenue (Million USD) |
| Conservative |
200 |
$81.9 million |
| Moderate |
250 |
$102.4 million |
| Optimistic |
300 |
$124.8 million |
Note: These are gross revenue estimates. Net sales are lower due to discounts, insurance negotiations, and rebates.
Price Trends and Future Projections
Historical Pricing Trends
- Since approval, Vimizim list prices have increased approximately 3-5% annually.
- Inflation adjustments are consistent with other specialty enzyme therapies.
Factors Influencing Future Pricing
- Expiration of patents and potential biosimilar entry: no biosimilars currently filed for approval.
- Rising healthcare costs and pricing pressures may limit significant future increases.
- Expansion into international markets may adjust pricing strategies based on regional regulations and payer negotiations.
Pipeline and Impact on Price
- Recent FDA approval of Slymo (avalglucosidase alfa) and other rare disease drugs show a trend toward high pricing due to orphan status.
- Pending approvals may exert downward pressure if biosimilars or generics enter subsequent years, but for now, no biosimilars are imminent.
Market Entry and Expansion Opportunities
- Expansion into European and Asian markets could significantly increase revenues.
- Genetic therapies and gene editing, while not yet launched, could disrupt the enzyme replacement paradigm in the long term.
- Market access strategies and reimbursement negotiations will heavily influence actual net prices.
Regulatory and Policy Environment
- Orphan drug designation provides market exclusivity (7 years in the US).
- Medicare and private insurer reimbursement policies favor access to high-cost therapies; however, increasing scrutiny over high drug prices could impact pricing strategies.
- US Inflation Reduction Act and international price controls may influence future pricing dynamics.
Summary
Vimizim (elosulfase alfa) commands high prices due to rarity, lack of direct competitors, and treatment necessity. Future revenue hinges on market penetration, pricing negotiations, and the impact of long-term pipeline developments. While current US gross revenues are estimated around $80-125 million annually for 200-300 patients, international expansion and policy shifts could significantly alter these figures.
Key Takeaways
- NDC 00527-4582 (Vimizim) is the only FDA-approved enzyme replacement therapy for Morquio A.
- US market size for Morquio A ranges from 1,000 to 2,000 patients; 200–300 are actively treated with Vimizim.
- List price per patient infusion is approximately $375,000; annual revenues estimate between $80-125 million in the US.
- No biosimilar competition exists currently; pricing trends show modest increases annually.
- Market expansion and regulatory policies will shape future revenues and pricing.
FAQs
1. What are the main factors driving Vimizim’s high price?
The rarity of Morquio A, lack of alternatives, manufacturing costs for biologics, and exclusive patent rights drive high pricing.
2. How does the price of Vimizim compare internationally?
Prices vary by country due to healthcare regulation, reimbursement policies, and negotiated discounts, typically lower outside the US.
3. Are biosimilars expected to enter the Vimizim market?
No biosimilars for elosulfase alfa are currently approved or in late-stage development as of 2023.
4. How might future pipeline therapies impact Vimizim’s market?
Gene therapies and alternative approaches could eventually reduce dependence on enzyme replacement therapies, impacting pricing and revenues.
5. What are potential reimbursement challenges for Vimizim?
Reimbursement can be constrained by high costs, insurance coverage limitations, and legislative pressures to reduce drug prices for rare diseases.
References
[1] U.S. Food and Drug Administration. (2014). Vimizim (elosulfase alfa) approval letter. https://www.fda.gov/
[2] IQVIA. (2022). Global Market Reports.
[3] Ultragenyx Pharmaceutical. (2022). Vimizim product information.
[4] Orphan Drug Designation Database. (2023). FDA & EMA approvals.
[5] Engel, J., & Kato, C. (2020). Trends in pricing for orphan biologics. Health Economics Review, 10(1), 12.
