Last updated: March 2, 2026
What is NDC 42806-0101?
NDC 42806-0101 refers to a specific drug product, which is identified by the National Drug Code (NDC). Based on the NDC database, this code corresponds to Mepsevii (vesiclesatase alfa), used for the treatment of mucopolysaccharidosis VII (MPS VII), a rare lysosomal storage disorder.
Market Overview
Indication and Patient Population
- MPS VII affects fewer than 200 patients in the U.S., classified as an ultra-rare disease.
- No current alternative treatments directly target MPS VII; enzyme replacement therapy (ERT) is the standard approach.
- The patient base is segmented into pediatric and adult populations, with diagnosis often delayed due to rarity and nonspecific symptoms.
Market Size
Table 1. Estimated Patient Population in the U.S. (2023)
| Population Segment |
Estimated Patients |
Source/Notes |
| Pediatric (under 18) |
150 |
Based on clinical case reports |
| Adult (18+) |
50 |
Lower due to disease progression and detection gaps |
| Total |
200 |
Total estimated in the U.S. |
Market Drivers
- Unmet medical need due to lack of alternative therapies
- Advancements in diagnostics increasing early detection
- Potential approvals in Europe and other regions expanding access
Competitive Landscape
- Mepsevii remains the first FDA-approved enzyme replacement therapy for MPS VII (FDA, 2017)
- Limited off-label use of treatments approved for other MPS types, such as MPS I or II, due to differing enzyme deficiencies
- Pipeline candidates are limited, with no significant competitors announced as of 2023
Price Analysis and Revenue Projections
Pricing Overview
- List Price (U.S.): Approximately $375,000 annually per patient (per manufacturer disclosures, 2022)
- Reimbursement models vary; most payers negotiate discounts or utilization caps
- Price increases have been modest, around 3-5% annually, reflecting market dynamics
Cost and Reimbursement Factors
- The high cost bears on insurer negotiations, impacting net revenue
- Payers may restrict access based on diagnosis confirmation and dosage regimens
- Government programs, like Medicaid, negotiate discounts
Revenue Projections (2023–2028)
| Year |
Estimated Patients Treated |
Average Price per Patient |
Gross Revenue (millions USD) |
Assumptions |
| 2023 |
150 |
$375,000 |
$56.25 |
Market stabilizes at current price and coverage |
| 2024 |
165 |
$390,000 |
$64.35 |
Slight increase in patient uptake |
| 2025 |
180 |
$405,000 |
$72.9 |
Growing diagnosis and expanding access |
| 2026 |
195 |
$420,000 |
$81.9 |
Broader payer coverage, pipeline progress |
| 2027 |
200 |
$435,000 |
$87 |
Near-saturation of diagnosed patients |
| 2028 |
200 |
$450,000 |
$90 |
Price adjustments for inflation |
Revenue Considerations
- Net revenue likely lower due to negotiated discounts (~20% on list price)
- Patent and market exclusivity are expected to persist through at least 2030
- New competitor entries could pressure prices beyond 2028
Key Market Risks and Opportunities
- Risks: Diagnostic delays limit market penetration, payer resistance to high-cost therapies, potential emergence of biosimilars
- Opportunities: Expansion into Europe and Asia, development of combination therapies, increased awareness
Summary
The market for NDC 42806-0101 (Mepsevii) remains niche but has a high-price-yield profile owing to its ultra-rare disease indication. Revenue projections center around $60 million in 2023, with gradual increases driven by diagnoses and pricing adjustments. Risks include payer restrictions and competitive advances. Long-term growth depends on geographic expansion and pipeline developments.
Key Takeaways
- The U.S. market size is approximately 200 patients, with annual market potential around $60 million at current pricing.
- Price projections suggest modest increases, constrained by payer negotiations.
- Market entry in Europe and Asia could significantly expand revenue streams.
- Advocacy, early diagnosis, and pipeline innovation are pivotal to growth.
- Competitive pressures and potential biosimilars pose long-term risks.
FAQs
1. What factors influence the pricing of NDC 42806-0101?
Pricing is influenced by manufacturer list prices, payer negotiations, market demand, and development costs.
2. Are there any approved biosimilars or generics for this drug?
No biosimilars or generics are approved for Mepsevii as of 2023; patent exclusivity persists.
3. How does the rare disease status impact market dynamics?
It limits patient volume but supports high per-patient pricing and incentives.
4. What regulatory developments could alter market outlook?
Additional approvals outside the U.S. and rare disease grant extensions could expand the market.
5. How does payer reimbursement affect net revenue?
Reimbursement negotiations typically result in discounts, reducing net revenue relative to list prices.
References
[1] U.S. Food and Drug Administration. (2017). FDA approval letter for Mepsevii. https://www.fda.gov/drugs/resources-information-approved-drugs/mepsevii
[2] IQVIA. (2022). Pricing trends for orphan drugs.
[3] GlobalData. (2023). Rare disease drug market analysis.
[4] Orphanet. (2023). Mucopolysaccharidosis VII.
[5] Centers for Medicare & Medicaid Services. (2022). Reimbursement policies for rare disease treatments.
