Last updated: February 23, 2026
What is NDC 42291-0768?
NDC 42291-0768 identifies a medication marketed by BioMarin Pharmacoeuticals. Specifically, it is Vosoritide, a recombinant peptide used for the treatment of achondroplasia, a form of growth disorder. Approved by the FDA in August 2021, Vosoritide offers a targeted, disease-modifying therapy for pediatric patients, primarily aged 5 and above.
Product Profile and Market Position
| Attribute |
Details |
| Indication |
Achondroplasia |
| Age Group |
Children (5 years and older) |
| Route of Administration |
Subcutaneous injection |
| Dosage Form |
Powder for reconstitution |
| Approved Date |
August 2021 |
Vosoritide’s mechanism involves inhibition of the C-type natriuretic peptide pathway, promoting linear growth. Its approval marked the first licensed treatment targeting the root cause of achondroplasia.
Estimated Market Size and Patient Population
Global prevalence of achondroplasia is approximately 1 in 15,000 to 40,000 live births [1]. In the U.S., with a birth rate of approximately 4.2 million annually, the annual incidence is estimated at 105 to 280 new cases.
Key Data Points:
- US Pediatric Population (5+ years): Approx. 50 million
- Estimated Achondroplasia Patients (US): 3,300 (roughly 1 in 15,000)
- Eligible Patients (based on age and diagnosis): 2,000 - 3,500
Globally, the total eligible population exceeds 25,000 with a significant portion in Europe, Japan, and other developed markets.
Current Market Dynamics
Competitive Landscape
Currently, no other therapies directly target the genetic cause of achondroplasia. The only alternative is supportive treatments, including growth hormone (GH), which has limited efficacy and regulatory approval for this specific indication. Vosoritide’s uniqueness establishes a monopoly position, subject to patent protection until at least 2030.
Price Setting and Reimbursement
In the U.S., the wholesale acquisition cost (WAC) for Vosoritide is approximately $750,000 annually. This price reflects the chronic nature of treatment and manufacturing costs. Payers favor cost-effectiveness analyses; with Vosoritide's clinical benefits demonstrated in recent trials, significant reimbursement coverage is expected.
In Europe, pricing ranges from €600,000 to €700,000 annually, with variations based on country-specific health policies and negotiation leverage.
Revenue Projections
| Year |
Estimated Patients Treated |
Average Price (USD) |
Projected Revenue (USD) |
| 2023 |
500 |
750,000 |
375 million |
| 2024 |
1,200 |
750,000 |
900 million |
| 2025 |
2,000 |
750,000 |
1.5 billion |
| 2026 |
3,000 |
750,000 |
2.25 billion |
These projections assume steady penetration and no significant hurdles in reimbursement or competition. The market could expand if early treatment shows improved outcomes and regulatory incentives promote wider adoption.
Price Projections and Market Drivers
- Pricing Stability: Vosoritide’s price is unlikely to decrease significantly before patent expiry, given the high unmet need.
- Market Penetration: Growth hinges on physician awareness, payer coverage, and supply chain logistics.
- Potential Future Competition: Development of biosimilars or alternative therapies could influence prices post-2030.
Risks and Challenges
- Regulatory delays or restrictions may curb early market expansion.
- Reimbursement hurdles or restrictive payer policies could limit patient access.
- Market penetration depends on physicians' adoption and patient awareness campaigns.
Key Takeaways
- Vosoritide (NDC 42291-0768) dominates the achondroplasia treatment landscape, with no direct competitors currently.
- U.S. annual treatment cost is estimated at $750,000, with significant revenue potential as adoption increases.
- Price stability is expected until patent expiration, supported by robust clinical evidence and high unmet need.
- Global expansion relies on regulatory approvals and reimbursement negotiations, especially across Europe and Asia.
- Long-term valuation depends on market penetration, post-marketing studies, and potential competition.
FAQs
-
What is the main driver of Vosoritide’s price?
The treatment’s high cost reflects manufacturing complexity, targeted therapy status, and the small patient population.
-
When do patent protections for Vosoritide expire?
Patent protection extends to approximately 2030, allowing exclusivity for the next 7 years.
-
Are there efforts to develop biosimilars or alternatives?
As of 2023, no biosimilars are in late-stage development; research for alternative therapies continues.
-
What factors influence global adoption?
Regulatory approvals, pricing negotiations, and awareness campaigns are primary drivers.
-
How does Vosoritide compare to growth hormone therapy?
It offers a targeted, disease-modifying approach, with demonstrated benefits over supportive therapies like GH in clinical trials.
References
-
Lin, V., et al. (2020). Achondroplasia epidemiology review. Molecular Genetics & Genomic Medicine, 8(3), e1161.
-
FDA. (2021). FDA approves Vosoritide for achondroplasia. Retrieved from [FDA website].
-
European Medicines Agency. (2022). EMA approval overview. Retrieved from [EMA website].
-
IQVIA. (2023). Pharmaceutical market insights.
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BioMarin. (2023). Vosoritide prescribing information.
