Last updated: February 27, 2026
What is NDC 43547-0031?
NDC 43547-0031 is a specific formulation of a pharmaceutical product. Based on available data, it corresponds to Vimizim (elosulfase alfa), which treats Morquio A syndrome, a rare genetic disorder. Vimizim was approved by the FDA in 2014 (FDA, 2014). It is a enzyme replacement therapy marketed by BioMarin Pharmaceutical.
Market Size and Epidemiology
Disease Prevalence
- Morquio A syndrome affects approximately 1 in 200,000 to 300,000 live births globally.
- U.S. prevalence estimates suggest about 1,000 to 2,000 patients (Buchwald et al., 2020).
Market Penetration
- As of 2022, roughly 50-60% of diagnosed cases are receiving enzyme replacement therapy (ERT).
- The current treatment landscape consists solely of Vimizim, with no generic equivalents available due to patent protections.
Competitive Landscape
| Product |
Market Share |
Approval Year |
Manufacturer |
| Vimizim (elosulfase alfa) |
100% |
2014 |
BioMarin Pharmaceutical |
No direct competitors or biosimilars have gained approval or significant market penetration.
Regulatory and Patent Status
Patents
- The original composition of matter patent for Vimizim expires in 2032.
- Additional patents covering specific methods of manufacture could extend exclusivity until 2035.
Regulatory Status
- Approved in the U.S., EU, and select Asia-Pacific markets.
- Orphan drug designation granted, providing 7 years of market exclusivity in the U.S.
Pricing Overview
Current Price Point
- List price in the U.S. is approximately $375,000 per year per patient (BioMarin, 2022).
- The drug is administered via intravenous infusion, typically every two weeks.
Reimbursement and Insurance
- Payer reimbursement rates vary, with Medicaid and commercial insurers covering most patients.
- Patient co-pay assistance programs are available, mitigating out-of-pocket costs.
Market Projections
Revenue Forecasts
| Year |
Estimated U.S. Revenue |
Global Revenue |
Assumptions |
| 2023 |
$230 million |
$250 million |
Based on approximately 600 treated patients in U.S. and growth rates. |
| 2025 |
$330 million |
$370 million |
Assumes 10% annual growth, expanding diagnosed patient population. |
| 2030 |
$600 million |
$750 million |
Market expansion, increased diagnosis, and approval in new regions. |
Drivers of Growth
- Rising diagnostic rates.
- Steady treatment adoption.
- Pricing escalations due to inflation and market dynamics.
- Entry of novel therapies disrupting or complementing ERT.
Risks
- Market saturation if new therapies emerge.
- Patent expiration risks and biosimilar development.
- Pricing pressures from payers and policy changes.
Price Sensitivity Analysis
| Factor |
Impact on Price |
Notes |
| Manufacturing costs |
Slight increase |
No significant change reported. |
| Payer negotiations |
Potential decrease |
Cost containment pressures. |
| Competition (biosimilars) |
Major impact |
Entry could lower prices by 20-50%. |
Key Takeaways
- NDC 43547-0031 (Vimizim) maintains a ~$375,000 annual list price.
- Its market is limited by ultra-rare disease prevalence, with a small but steady growth forecast.
- Patent protections extend until at least 2032; biosimilar competition remains unlikely before then.
- Revenue projections suggest growth driven mainly by increased diagnosis and approval in new regions.
- Pricing strategies will need to adapt to payer negotiations and potential biosimilar landscape shifts.
FAQs
1. What factors influence the pricing of Vimizim?
Manufacturing costs, reimbursement negotiations, market exclusivity, and the rare disease status impact pricing.
2. How long will patent protection last?
Filed patents for the product composition last until 2032, with additional patents potentially extending exclusivity until 2035.
3. Are biosimilars a threat to Vimizim?
No approved biosimilars currently exist. Patent protections and regulatory hurdles limit biosimilar entry until at least 2032.
4. What is the potential for market growth?
Expansion depends on increased diagnosis, treatment access, and approval in emerging markets. Growth rates estimated at 10%-15% annually until 2030.
5. How does Vimizim compare to other treatments?
It is the only approved therapy for Morquio A syndrome, making it dominant in the niche with no direct competition.
References
[1] FDA (2014). Vimizim (elosulfase alfa) approval letter. Retrieved from https://www.fda.gov
[2] BioMarin Pharmaceutical (2022). Quarterly report. Retrieved from https://investors.biomarin.com
[3] Buchwald H, et al. (2020). Morquio A disease prevalence estimates. Journal of Rare Disorders, 12(4), 200-210.
