Last updated: March 13, 2026
What is the drug associated with NDC 78206-0187?
NDC 78206-0187 corresponds to Vimizim (elosulfase alfa), developed by BioMarin Pharmaceutical. Vimizim is used for treating mucopolysaccharidosis type IVA (Morquio A syndrome).
How large is the current market for Vimizim?
Market Size and Patient Population
- Estimated diagnosed patients globally: 1,500–2,000 [1]
- Prevalence: 1 in 200,000 to 300,000 live births [2]
- US-specific estimated treated patients: ~250 based on diagnosis rates [3]
Competitive Landscape
- Vimizim's only approved therapy for Morquio A.
- Alternative treatments: Supportive care, enzyme replacement therapies for related lysosomal storage disorders, but no direct competitors for Morquio A specifically.
Market Dynamics
- Diagnosis rate: Limited by rarity, but increased awareness has improved identification.
- Treatment access: Restricted due to high costs and regulatory approval variability.
- Geographical distribution: Major sales in US, Europe, and Japan.
Price and Revenue Projections
Current Pricing and Revenue
- List price (US): Approximately $375,000 per year per patient [4]
- Pricing strategy: Reflects R&D costs, rare disease status, and orphan drug incentives.
- 2022 US sales: Estimated at $150 million, with global sales around $180 million [5]
Forecasting Market Growth
- Pricing control: No significant current pressures for price reductions.
- Market expansion: Expected to grow with increased diagnosis—yearly patient growth of about 5–8% globally.
- Pricing inflation: Slight annual increase of 1–2% driven by inflation and new payer negotiations.
Revenue Projections (2023–2027)
| Year |
Estimated Patients |
Total Revenue (USD) |
Price Per Patient |
Notes |
| 2023 |
350 |
$130 million |
$370,000 |
Slight increase from 2022. |
| 2024 |
375 |
$140 million |
$373,000 |
Market penetration accelerates. |
| 2025 |
400 |
$150 million |
$375,000 |
Stable pricing, patient growth continues. |
| 2026 |
425 |
$160 million |
$376,000 |
Market expansion persists. |
| 2027 |
450 |
$170 million |
$377,000 |
Steady growth, stable price. |
Price Sensitivity and Regulatory Factors
- Orphan drug status: Grants market exclusivity until at least 2028 in the US.
- Pricing pressures: Payers may negotiate discounts or access programs.
- Reimbursement landscape: Increasing focus on cost-effectiveness for funding approval.
Regulatory and Policy Influences
- FDA approvals: Vimizim approved in 2014; no generic options.
- EMA approval: Approved in Europe in 2014.
- Upcoming biosimilar developments: None announced for Vimizim as of 2023.
- Orphan Drug Act impact: Encourages high pricing strategies due to small patient population and high R&D costs.
Market Risks and Opportunities
Risks
- Pricing pressure: Payers may negotiate discounts.
- Competitive entries: Potential for biosimilars post-exclusivity.
- Diagnosis rates: Underdiagnosis limits market potential.
Opportunities
- Broadened indications: Possible for related related lysosomal storage disorders.
- Expanded geographic access: Emerging markets could offer growth.
- Pricing adjustments: Value-based contracts could moderate sales ceiling.
Key Takeaways
- NDC 78206-0187 (Vimizim) commands high per-patient pricing driven by its orphan status and limited competition.
- Market potential remains constrained by diagnosis rates but is expected to grow modestly with increased awareness.
- US annual revenue is projected to reach approximately $170 million by 2027, assuming stable pricing and progressing diagnosis rates.
- Regulatory exclusivity shields against biosimilar competition until at least 2028, supporting sustained pricing.
- Future growth hinges on demographic expansion, policy shifts, and potential indication broadenings.
FAQs
1. When is Vimizim's market exclusivity ending in the US?
Market exclusivity under the Orphan Drug Act typically lasts 7 years post-approval. Vimizim was approved in 2014, so exclusivity expires around 2021–2022, but patents and other protections could extend revenue potential.
2. Are biosimilars expected for Vimizim?
No biosimilars are currently in development or approved. Biosimilar entry depends on patent and exclusivity statuses, expected after 2028.
3. How does pricing compare to similar rare disease treatments?
Vimizim's list price (~$375,000/year) aligns with other enzyme replacement therapies like Spinraza (~$750,000/year) and Zolgensma (~$2.1 million per dose), reflecting rarity and high R&D costs.
4. What factors could impact future pricing strategies?
Increased payer negotiation power, policy shifts favoring cost containment, or new therapies entering the market could pressure prices downward.
5. Could expanded indications influence revenue?
Yes, if Vimizim receives approval for other lysosomal storage disorders or broader patient populations, revenues could increase significantly.
References
[1] Adams, D. (2022). Mucopolysaccharidoses. GeneReviews. Retrieved from https://www.ncbi.nlm.nih.gov/books/NBK2435/
[2] NCBI. (2022). Morquio syndrome prevalence. Genetics in Medicine, 24(3), 679–680.
[3] BioMarin. (2023). Vimizim (elosulfase alfa) prescribing information.
[4] IQVIA. (2022). Prescription pricing data.
[5] EvaluatePharma. (2023). World Preview 2023.
