Last updated: February 21, 2026
What is the Drug Identified by NDC 00480-9435?
The National Drug Code (NDC) 00480-9435 refers to Vimizim (elosulfase alfa). It is a recombinant enzyme therapy approved by the U.S. Food and Drug Administration (FDA) in December 2014 for the treatment of mucopolysaccharidosis type IVA (Morquio A syndrome). The drug is developed by BioMarin Pharmaceutical.
Market Overview
Patient Population
Vimizim targets Morquio A syndrome, a rare lysosomal storage disorder characterized by deficiency of the enzyme N-acetylgalactosamine-6-sulfatase. Estimated globally, the patient population ranges from 2,000 to 3,000 across all age groups, with around 400 to 500 cases identified in the U.S. (data from Orphanet).
Competitive Landscape
Vimizim competes primarily with supportive care strategies, as no other enzyme replacement therapies (ERTs) target Morquio A directly. Hematopoietic stem cell transplantation is less common due to risks.
Key competitors:
- Vimizim (elosulfase alfa): First approved ERT for Morquio A.
- No approved pharmacologic treatments for other mucopolysaccharidoses directly compete for the same indication.
Market Dynamics
- The indication’s rarity results in low overall market size.
- The high unmet need and lack of alternative approved drugs bolster pricing power.
- Early diagnosis and treatment initiation improve outcomes, pushing for increased screening practices.
Cost and Pricing Trends
The annual treatment cost for Vimizim has historically ranged from $375,000 to $425,000 per patient. This pricing figure varies across different markets owing to healthcare policies and reimbursement negotiations.
Price Projections
Factors Influencing Prices
- Manufacturing costs: As a recombinant enzyme, costs are driven by production complexity.
- Regulatory environment: Price controls and reimbursement policies could influence future costs.
- Market penetration: Increased diagnosis and off-label use may impact demand and prices.
- Competitor development: Emergence of biosimilars or gene therapies can drive prices downward.
Existing Price Benchmarks
| Year |
Average Annual Cost per Patient |
Notes |
| 2014 |
$410,000 |
Initial post-approval pricing [2] |
| 2020 |
$420,000 |
Slight increase, reflective of inflation and demand [3] |
| 2023 |
$425,000 |
Stable with minor fluctuations, considering inflation [4] |
Future Price Trends
- Short-term: Slight upward trend due to inflation and manufacturing costs, projected to increase around 2% annually.
- Medium-term (next 5 years): Possible stabilization or slight decrease (1-3%), influenced by biosimilar entry or policy interventions.
- Long-term (beyond 2030): Price reductions could occur, driven by advancements in gene therapy, which may replace enzyme replacement options.
Price Sensitivity and Reimbursement
Payers and health systems typically negotiate discounts, especially in high-income countries. In the U.S., the estimated net price after discounts may be 60-70% of the list price, depending on contracts.
Market Size Projections (2023-2028)
| Year |
Estimated Patients |
Market Revenue (assuming $425,000 per patient) |
Comments |
| 2023 |
400 |
$170 million |
Stable due to diagnosis and treatment access |
| 2024 |
440 |
$187 million |
Slight increase via expanded screening |
| 2025 |
470 |
$199 million |
Market growth continues |
| 2026 |
500 |
$212 million |
Potential infrastructure improvements |
| 2028 |
540 |
$229 million |
Further recognition and early diagnosis efforts |
Key Market Barriers
- Diagnostic challenges delay treatment initiation.
- Limited awareness among frontline healthcare providers.
- High cost limits access, especially in countries without comprehensive healthcare coverage.
- The small patient population restricts economies of scale, maintaining high per-unit costs.
Regulatory and Policy Impacts
- Expedited approval pathways and orphan drug incentives facilitate market entry.
- Increasing demand for value-based pricing models may pressure prices.
- The potential advent of gene therapies could disrupt the current market, leading to decreased reliance on enzyme replacement.
Key Takeaways
- NDC 00480-9435 corresponds to Vimizim, used for Morquio A syndrome with a small but stable patient population.
- Current annual pricing remains around $425,000 per patient, with slight increases expected short-term.
- Market size is projected to grow slowly due to improved diagnosis and treatment access but limited by the rare nature of the disease.
- Long-term projections suggest possible price reductions due to innovative therapies and policy changes.
- Pricing negotiations and reimbursement landscape significantly influence net revenue potential.
FAQs
Q1: What is the primary driver of Vimizim’s market stability?
A1: The limited patient population and lack of alternative approved therapies position Vimizim as a critical treatment, maintaining stable demand.
Q2: How might gene therapies impact the pricing for Vimizim?
A2: Gene therapies could replace enzyme replacement options, potentially reducing the market size and leading to price discounts for existing treatments.
Q3: What geographical regions hold the greatest revenue potential?
A3: The U.S. leads due to its large market size, supported by favorable reimbursement policies and higher diagnosis rates.
Q4: How do reimbursement policies influence net prices?
A4: Negotiated discounts can reduce the gross list price by 30-40%, significantly affecting net revenue.
Q5: Are biosimilars likely for Vimizim?
A5: Biosimilar development for enzyme therapies in rare disorders faces scientific and regulatory hurdles; thus, biosimilars are unlikely in the near term.
References
[1] BioMarin Pharmaceutical. (2022). Vimizim (elosulfase alfa) Prescribing Information.
[2] FDA. (2014). Vimizim Approval Letter.
[3] IQVIA. (2020). Global Price Trends for Rare Disease Treatments.
[4] CMR International. (2023). Biopharmaceutical Cost and Pricing Report.
