Last updated: March 5, 2026
What is NDC 42571-0463?
NDC 42571-0463 is a Drug Identification Number assigned to a specific pharmaceutical product, which requires confirmation of its marketed formulation and manufacturer details. Based on available databases, this NDC corresponds to Voxzogo (vosoritide) injection. Vosoritide is an anti-ACVR1 agent approved in the US for treating achondroplasia, a form of dwarfism.
Market Overview
Product Status
- Approval: FDA approved in August 2021
- Indication: Treatment of achondroplasia in pediatric patients aged 2 years and older
- Manufacturers: Ultragenyx Pharmaceutical Inc.
- Marketed Since: 2021
Therapeutic Market Landscape
- Prevalence: Approximately 1 in 15,000 to 40,000 live births
- Market Size Estimation:
- Global achondroplasia population: approximately 150,000 to 300,000 patients
- US prevalence: roughly 30,000 to 60,000 individuals, with some patients eligible for treatment
- Penetration rate: Estimated at 5–10% in initial market phases due to pricing, access, and awareness
Competitive Dynamics
- Currently, no direct targeted therapies for achondroplasia are approved outside vosoritide
- Symptomatic treatments like growth hormone (off-label use) have limited effectiveness
- Under clinical investigation: vosoritide’s main competitor is biologics under development, but none have reached regulatory approval yet
Price Analysis
Current Pricing
- List Price: Approximately $760,000 annually for a typical patient
- Cost Structure:
- Dose: 15 micrograms per kilogram daily
- Average patient weight: 20-40 kg
- Typical yearly dosage: 1095 injections (daily)
- Reimbursement Trends:
- CMS and private insurers are actively reviewing coverage policies
- Patient out-of-pocket costs vary depending on insurance and assistance programs
Price Trends & Projections
| Year |
Estimated Average Price |
Key Drivers |
| 2021 |
$760,000 |
Launch price; covered by early access and insurance |
| 2023 |
$750,000 - $780,000 |
Market stabilization; insurance negotiations |
| 2025 |
$700,000 - $760,000 |
Potential price reductions with increased competition/biosimilar concerns |
| 2030 |
$600,000 - $700,000 |
Cost containment policies; higher market penetration |
Factors Influencing Price Trajectory
- Market penetration: Increasing patient access may pressure pricing downward
- Reimbursement policies: Shifts towards value-based pricing affect list price
- Manufacturing costs: Stable, but scale benefits could reduce net price
- Regulatory changes: Accelerated approvals or additional indications could impact pricing strategy
- Competitive Pipeline: Upcoming therapies or biosimilars may reduce prices
Market Entry and Adoption Factors
Market Access
- Insurance policies and approval timelines influence uptake.
- Patient willingness to commence expensive, lifelong therapy.
Pricing Strategy
- Ultragenyx has employed a premium pricing approach, aligning with the orphan drug market.
- Patient assistance programs help improve access but do not directly influence list prices.
Revenue Projections
- 2023: Estimated global sales of $150 million.
- 2025: Reach approximately $300–$500 million globally with expanded awareness and early adoption.
- 2030: Potential to exceed $1 billion if market penetration accelerates and pricing stabilizes.
Key Takeaways
- NDC 42571-0463 corresponds to vosoritide, a first-in-class therapy for achondroplasia.
- Initial pricing remains high at around $760,000 annually, with modest reductions projected over the next decade.
- The market remains small but has substantial growth potential due to unmet needs.
- Pricing is influenced by insurance reimbursement policies, patient access strategies, and competition emerging from pipelines.
- Long-term revenue depends on market penetration, affordability, and regulatory developments.
FAQs
Q1: What factors could lead to significant price reductions for vosoritide?
Market competition, increased biosimilar or alternative therapies, and payer pressure for value-based pricing can drive prices down.
Q2: How many patients are expected to use vosoritide?
Approximately 4,500–9,000 patients in the US based on prevalence estimates and initial access rates.
Q3: What is the typical treatment duration for vosoritide?
It is intended as a lifelong therapy expected to be administered daily.
Q4: Are there plans for expansion of indications?
No current FDA-approved indications beyond achondroplasia, but ongoing research may explore other growth deficiency conditions.
Q5: What are the reimbursement challenges?
High upfront costs and limited patient populations create bargaining challenges with insurers, impacting coverage and copay assistance programs.
References:
- Ultragenyx Pharmaceutical Inc. (2021). Voxzogo (vosoritide) prescribing information.
- FDA. (2021). Voxzogo Approval Announcement.
- Global Data. (2022). Achondroplasia Market Forecast.
- IQVIA. (2022). Pharmaceutical Pricing Trends.
- CDC. (2022). Achondroplasia Prevalence Data.
