Last updated: February 23, 2026
What is the drug associated with NDC 00713-0865?
NDC 00713-0865 is the product "Vimizim" (elosulfase alfa), indicated for Morquio A syndrome (Mucopolysaccharidosis IVA). It is a enzyme replacement therapy developed by BioMarin Pharmaceutical.
Market Overview
Disease Background and Treatment Adoption
- Morquio A syndrome is a rare, inherited lysosomal storage disorder caused by a deficiency of the enzyme N-acetylgalactosamine-6-sulfatase.
- Prevalence estimates: approximately 1 in 200,000 to 300,000 live births globally.
Treatment Landscape
| Company |
Product |
Indication |
Approval Date |
Market Share (%) |
| BioMarin |
Vimizim |
Morquio A syndrome |
2014 |
100 (initial market) |
- No direct competitors approved specifically for Morquio A. Off-label treatments focus on symptom management.
Market Penetration & Challenges
- Slow diagnosis rates delay treatment initiation.
- Limited patient populations restrict market size.
- Manufacturing complexities for biologics impact supply chain costs.
Price and Revenue Estimates
Historical Pricing
- List Price (average in the U.S.): approximately $375,000 per year per patient (source: Medicare Part B data, 2020).
Cost Breakdown
| Cost Component |
Estimated Cost ($) |
Details |
| Raw materials |
15-20% of list price |
High-quality recombinant protein production |
| Manufacturing |
25-30% of list price |
Biologics manufacturing complexity |
| Distribution & supply chain |
10-15% |
Cold chain logistics |
| Sales & Marketing |
10% |
Limited, targeted marketing |
| Profit Margin |
15-20% |
Industry average for biologics |
Price Projections (Next 5 Years)
| Year |
Projected List Price |
Rationale |
| 2023 |
$375,000 |
Current market established |
| 2024 |
$385,000 |
Annual inflation adjustment (~2.7%) |
| 2025 |
$395,000 |
Price normalization post any potential biosimilar entry |
| 2026 |
$405,000 |
Continued inflation, potential payer negotiations |
| 2027 |
$415,000 |
Anticipated increase if demand sustains |
Impact on Revenue
- Total U.S. patient population: roughly 400-600 patients (approximate, due to diagnostic delays and underdiagnosis).
- Revenue estimate for 2023: 500 patients × $375,000 = $187.5 million.
- Projected growth driven by increased awareness and diagnosis rates could raise the number of treated patients by 5-10% annually.
Patent and Regulatory Risks
- BioMarin’s patent portfolio provides exclusivity until at least 2030.
- Biosimilar entry expected post-patent expiration, but timelines remain uncertain.
- Pricing regulation pressures could impact revenue margins.
Market Constraints & Opportunities
Constraints
- Small patient population limits revenue scale.
- High manufacturing costs limit pricing flexibility.
- Insurance and government reimbursement policies influence net pricing.
Opportunities
- Expansion into emerging markets where approval policies are evolving.
- Development of improved formulations or biosimilars.
- Increased diagnostic campaigns expanding patient pool.
Summary of Key Indicators
| Metric |
Status |
Sources |
| U.S. Patient Estimate |
400-600 patients |
[1] |
| List Price (U.S.) |
$375,000 per year |
[2] |
| Expected 5-year Price Trend |
+2-3% annually |
Industry average inflation |
| Revenue (2023) |
$187.5 million |
Calculated from patient estimate × price |
| Patent Expiry |
~2030 |
BioMarin patents (2023 filings) |
Key Takeaways
- NDC 00713-0865 (Vimizim) operates within a small, high-cost niche market.
- Price projections increase with inflation, maintaining stable revenue streams barring biosimilar entry.
- Market growth depends on diagnosis rates, reimbursement policies, and regulatory landscape.
- Manufacturing complexity and patent protections strongly influence pricing power and revenue forecasts.
FAQs
1. What factors influence Vimizim's pricing strategy?
List price adjustments are driven by inflation, manufacturing costs, reimbursement negotiations, and competitive landscape.
2. How is the small patient base impacting revenue potential?
Limited patient numbers restrict total sales volume, making price and market access critical to revenue.
3. When are biosimilars expected, and how will they affect the market?
Biosimilars may enter around 2030 post-patent, potentially reducing prices and market share.
4. Are there emerging markets for this drug?
Yes, countries with advancing healthcare infrastructure and evolving approval pathways present opportunities.
5. What are the main risks to revenue growth?
Patent cliffs, biosimilar competition, pricing pressure, and diagnostic delays.
References
[1] Orphanet. (2022). Morquio syndrome (Mucopolysaccharidosis IVA). Available at: https://www.orpha.net/consor/cgi-bin/OC_Exp.php?Lng=EN&Expert=325
[2] BioMarin Pharmaceutical Inc. (2020). Vimizim Product Information. Available at: https://www.biomarin.com/our-products/vimizim
