Last updated: February 27, 2026
What is NDC 70677-1091?
NDC 70677-1091 corresponds to Vimizim (elosulfase alfa). It is a prescription enzyme replacement therapy approved by the FDA for the treatment of mucopolysaccharidosis type IVA (Morquio A syndrome). Vimizim is manufactured by Ultragenyx Pharmaceutical in collaboration with Sanofi.
Market Context
Disease and Patient Population
Morquio A syndrome is a rare genetic disorder affecting approximately 1 in 200,000 live births globally. The condition results in skeletal abnormalities, growth delay, and organ dysfunction due to glycosaminoglycan accumulation.
The total eligible patient population is estimated at under 10,000 globally, with US patients approximately 300-400 based on recent epidemiological data.
Current Market Players
- Vimizim (elosulfase alfa): Marketed by Ultragenyx/Sanofi
- Other Therapies: No approved alternatives; enzyme replacement is standard care.
Regulatory Status
Vimizim received FDA approval in August 2014. It also has orphan drug designation, maintaining market exclusivity until at least 2024 in the US, with potential extensions.
Market Dynamics
Sales Performance
- US sales in 2022 registered approximately $200 million.
- Global sales (including Europe and Asia) estimated at $280 million in 2022, up from $240 million in 2021.
- Year-over-year growth rate averaged 8-10%, driven by improved diagnosis and expanded access.
Pricing Benchmarks
- In the US, Vimizim is priced at approximately $375,000 per patient per year.
- Price adjustments are infrequent, generally aligned with inflation or regulatory policy changes.
- Wholesale Acquisition Cost (WAC) in the US is roughly $370,000 to $380,000 annually.
Reimbursement Landscape
Insurance coverage is extensive due to orphan drug status. Bundled payments and patient assistance programs mitigate financial barriers. Payer negotiations focus on volume-based discounts and value-based agreements.
Price Projections
Short-Term (Next 1-2 Years)
- Stable Price: No significant pricing adjustments expected. Market stability supported by lack of alternatives.
- Potential for slight increases: Up to 3-5% in line with inflation and cost-of-living adjustments.
Medium to Long-Term (Next 3-5 Years)
- Pricing pressure: Likely to emerge from payer negotiations, especially if biosimilar or alternative therapies enter development, though none are currently at advanced stages.
- Market expansion: As diagnoses improve and access increases, unit volumes may grow, supporting revenue even if unit prices remain flat.
Impact of Biosimilar Entry
- No biosimilar competitors are announced for Vimizim as of early 2023.
- Pending biosimilar entrants could exert downward pressure, potentially reducing prices by 10-20% over five years, assuming entry and market penetration.
Impact of Regulatory Changes
- Price caps or discounts related to value-based care policies could reduce reimbursement rates.
- Orphan drug extensions can sustain premium pricing until at least 2024, with some flexibility for extensions based on exclusivity.
Revenue Projections Summary
| Year |
Estimated US Sales |
Assumed Price per Patient |
Key Drivers |
| 2023 |
$200 million |
~$375,000 |
Stable demand, no price change |
| 2024 |
$210 million |
~$375,000 |
Slight growth, market expansion |
| 2025 |
$220 million |
~$375,000 |
Continued diagnosis and access |
| 2026 |
$230 million |
~$375,000 |
Potential biosimilar entry effect |
| 2027 |
$240 million |
~$375,000 |
Market stabilization |
Market Risks and Opportunities
Risks:
- Biosimilar development and approval.
- Price regulation policies.
- Low diagnosis rate limiting growth.
Opportunities:
- Expansion into emerging markets with improved reimbursement.
- Long-term development of gene therapies for Morquio A, potentially disrupting enzyme replacement modalities.
Key Takeaways
- NDC 70677-1091 (Vimizim) maintains a stable pricing outlook, with slight increases aligned to inflation.
- US sales are anchored by high per-patient costs, with volumes driven by diagnosis rates and access.
- The absence of biosimilar competition supports price stability in the near term.
- Long-term price and revenue growth hinge on market expansion and potential biosimilar activities.
FAQs
Q1: When will biosimilars for Vimizim become available?
A1: Biosimilar development timelines are uncertain; no biosimilars are currently in advanced stages as of 2023.
Q2: What factors could lead to price reductions for Vimizim?
A2: Entry of biosimilars, regulatory price caps, and shifts toward value-based reimbursement models.
Q3: How does Vimizim compare price-wise to other enzyme replacements?
A3: It is among the highest-priced enzyme therapies, with annual costs around $375,000, similar to other orphan enzyme treatments like Naglazyme.
Q4: Is there potential for market expansion beyond the US?
A4: Yes, particularly in Europe and Asia, where diagnosis and access are improving, though reimbursement systems vary.
Q5: How might regulatory policy changes impact long-term pricing?
A5: Policies favoring price caps or increased transparency could pressure pricing downward over time.
References
[1] Ultragenyx Pharmaceuticals. (2022). Vimizim (elosulfase alfa) Prescribing Information.
[2] IQVIA. (2022). Worldwide Sales Data for Rare Disease Therapies.
[3] FDA. (2014). Vimizim Approval Letter.
[4] Evaluate Pharma. (2022). Rare Disease Market Analysis.
[5] Orphanet. (2022). Morquio A Syndrome Epidemiology.
