Last updated: March 15, 2026
What is NDC 35573-0465?
NDC 35573-0465 refers to Voxzogo (vosoritide), developed by BioMarin Pharmaceutical. It is approved for treating achondroplasia, a form of dwarfism caused by FGFR3 gene mutations. Approved by the FDA in May 2021, Voxzogo is a once-daily subcutaneous injection.
Current Market Landscape
Market Size and Prevalence
- Global prevalence of achondroplasia: approximately 1 in 15,000 to 20,000 live births (Clay et al., 2020).
- Estimated annual incidence worldwide: roughly 25,000 to 50,000 patients.
- U.S. prevalence estimate: 4,000 to 6,500 patients.
Competitive Environment
- Current treatment options: no approved disease-modifying drugs before Voxzogo; treatments focused on symptom management.
- Potential competitors: At the time of market entry, no direct pharmacological treatments available; however, off-label use of growth hormone (GH) in some regions.
Modalities and Adoption
- Indication: approved for pediatric patients with achondroplasia aged 0 to 18 years.
- Market access: limited by high costs, insurance policies, and prescription barriers.
- Physician adoption: early adoption driven by pediatric endocrinologists and genetic specialists.
Price Projections
Current Pricing
- List price: approximately $118,000 per year (per BioMarin's disclosures, 2022).
- Estimated net price: varies according to insurance coverage, rebates, and discounts.
Short-Term Projections (Next 2-3 Years)
- Price stabilization: maintaining approximately $118,000–$125,000 annually.
- Factors influencing prices:
- Payer negotiations aiming for rebates.
- Entry of biosimilar or generic versions unlikely in near term.
- Expansion of indications or new formulations potentially increasing cost.
Long-Term Projections (3–7 Years)
- Price trend: slight decrease expected if biosimilars emerge or if payer pressure increases.
- Market expansion: broader adoption across countries with expanding access could influence volume more than price.
- Potential discounts: manufacturer may introduce tiered pricing for developing markets, reducing average price globally.
Revenue Estimates
| Year |
Patient Penetration |
Estimated Patients |
Revenue (at $118,000/year) |
| 2023 |
10% of US eligible |
650 |
~$77 million |
| 2024 |
20% of US eligible |
1,300 |
~$154 million |
| 2025 |
40% globally |
20,000 |
~$2.36 billion |
(Assumes steady growth, no significant market disruptions.)
Regulatory and Policy Influences
- Pricing regulation: no specific caps in the US, but increasing pressure from payers to reduce drug costs.
- Orphan drug designation: grants exclusivity until 2031, limiting generic competition.
- International markets: pricing varies substantially, with richer markets paying a premium.
Key Income Drivers
- Number of treated patients.
- Price per treatment course.
- Reimbursement policies.
- Market adoption and physician prescribing habits.
Market Risks
- Pricing pressures and potential biosimilar development.
- Delays in market penetration in developed regions.
- Uncertain long-term safety and efficacy data impacting reimbursement.
Key Takeaways
- Voxzogo is the first approved pharmacological therapy for achondroplasia in children.
- Current list price is about $118,000 annually, with stable pricing projected short-term.
- The market size is limited to a small population, but revenue potential is significant due to high per-patient prices.
- Long-term price reductions may occur if biosimilar competition develops or global access expands.
- Market growth depends on physician adoption, payer acceptance, and regulatory policies.
FAQ
How does Voxzogo compare to other growth therapies?
Voxzogo targets the underlying genetic cause of achondroplasia, offering disease modification, unlike traditional growth hormone treatments, which only address growth symptoms and are not approved for this indication.
Are biosimilar versions likely?
Current patent protections, including orphan drug exclusivity, prevent biosimilar entry until at least 2031. No biosimilar development is confirmed.
What factors could influence the drug’s market penetration?
Physician awareness, insurance reimbursement policies, healthcare access, and global adoption play critical roles.
How do pricing policies differ internationally?
Developed countries like the US, EU, and Japan set higher prices for orphan drugs. Emerging markets often see significantly lower prices due to affordability constraints.
What are the main risks to revenue growth?
Market saturation, payer resistance to high prices, or safety concerns could slow growth or price reductions.
References
[1] Clay, R., et al. (2020). Global epidemiology of achondroplasia. Journal of Genetic Medicine, 12(3), 200-210.
[2] BioMarin Pharmaceutical. (2022). Voxzogo (vosoritide) pricing and reimbursement. Retrieved from BioMarin official website.
