Last updated: February 27, 2026
What is NDC 46122-0416?
NDC 46122-0416 corresponds to Zolgensma (onasemnogene abeparvovec-xioi), a gene therapy approved by the FDA in May 2019 for treating spinal muscular atrophy (SMA) in pediatric patients under two years old. It is administered as a one-time intravenous infusion.
Market Size and Demographics
SMA is a rare genetic disorder affecting approximately 1 in 10,000 to 11,000 live births globally. In the United States, about 400 new cases are diagnosed annually.
Estimated Patient Population
| Region |
Estimated Annual SMA Patients < 2 Years |
Market Penetration (2022) |
Total Treated Patients (2022) |
| US |
400 |
55% |
220 |
| Europe |
1000 (total in EU) |
40% |
400 |
| Rest of World |
300 |
10-20% |
50-60 |
| Total |
1700 |
~50-55% |
~670-680 |
Market penetration is driven by approval status, reimbursement coverage, and physician awareness.
Competitive Landscape
Key Therapies
- Zolgensma: Gene therapy, one-time treatment.
- Spinraza (nusinersen): FDA approved since 2016, administered periodically.
- Evrysdi (risdiplam): FDA approved in 2020, oral administration.
Market Shares (2022 estimates)
| Therapy |
Market Share |
Annual Revenue (USD millions) |
Dosing/Administration Mode |
| Zolgensma |
35% |
1,000 |
Single infusion |
| Spinraza |
45% |
1,300 |
Intrathecal injections, quarterly or biannual |
| Evrysdi |
20% |
500 |
Oral daily |
Pricing Overview
- Zolgensma: Listed at $2.1 million per dose.
- Spinraza: $750,000 initial dose, then $375,000 every four months.
- Evrysdi: Approximate annual cost $340,000.
Pricing Trends and Projections
Historical Price Movement
| Year |
Zolgensma (per dose in USD) |
Spinraza (annual in USD) |
Evrysdi (annual in USD) |
| 2019 |
$2.125 million |
$750,000 |
- |
| 2020 |
$2.125 million |
$750,000 |
$340,000 |
| 2022 |
$2.125 million |
$750,000 |
$340,000 |
Since launch, Zolgensma price has remained stable. Reimbursement negotiations focus on value-based agreements rather than price reductions.
Future Price Projections (2024 - 2028)
- Inflation-adjusted stability: Prices are expected to remain stable due to high value and limited competition.
- Market expansion effect: Broader access in emerging markets may pressure prices, but current pricing models prioritize cost recovery and innovation recoupment.
| Projected Price Range (USD) |
Year |
Zolgensma |
Spinraza |
Evrysdi |
| 2024 |
$2.1 million |
$750,000 |
$340,000 |
| 2028 |
$2.1 million |
$750,000 |
$340,000 |
Market access costs and negotiations could influence actual reimbursement levels. Cost-effectiveness studies support Zolgensma's premium pricing, citing its curative potential.
Revenue Forecasts
US Market (2023-2028)
| Assuming a steady annual patient treatment increase of 10% |
Year |
Treated Patients (US) |
Revenue (USD millions) |
| 2023 |
220 |
462 |
| 2024 |
242 |
508 |
| 2025 |
266 |
558 |
| 2026 |
292 |
613 |
| 2027 |
321 |
675 |
| 2028 |
353 |
743 |
Global Market (2023-2028)
| Applying a proportionate increase based on estimated patient populations: |
Year |
Total Revenue (USD millions) |
| 2023 |
750 |
| 2024 |
830 |
| 2025 |
920 |
| 2026 |
1,020 |
| 2027 |
1,130 |
| 2028 |
1,250 |
Pricing stability and improved access are vital assumptions underpinning forecasts.
Key Regulatory and Policy Factors
- Reimbursement: High upfront costs provoke payor resistance; value-based contracts are evolving.
- Market Entry: Pending approval in Asian markets; biosimilar or follow-on gene therapies may develop within a decade.
- Policy Trends: Increasing emphasis on early diagnosis and newborn screening enhances market penetration.
Risks to Price and Market Projections
- Cost Containment Policies: Possible price caps or discounts.
- Technological Disruption: Emerging gene editing or RNA therapies could alter the landscape.
- Pricing Disputes: Reimbursement challenges could lead to slower adoption.
Summary
| Aspect |
Insight |
| Market size |
Approximately 1,700 new pediatric SMA cases globally annually |
| Current price |
$2.1 million per dose in the US; stable since 2019 |
| Revenue prospects |
US market reaching ~$743 million by 2028, global ~$1.25 billion |
| Competitive position |
Dominates as the only approved gene therapy; competing therapies maintain significant market share in non-gene therapy segments |
| Price trend |
Remains stable, with limited downward pressure expected |
Key Takeaways
- Zolgensma maintains premium pricing driven by its one-time curative profile.
- Market penetration will be influenced by reimbursement policies, diagnosis rates, and emerging therapies.
- Revenue projections are conservative, assuming continued stable pricing and increasing treatment rates.
- Regulatory developments in emerging markets could unlock additional growth.
- Cost management strategies from payors may impact future pricing and access.
FAQs
-
What factors influence Zolgensma’s pricing stability?
Its curative efficacy, limited competition, and negotiations centered on value-based pricing sustain current levels.
-
How does Zolgensma compare with Spinraza and Evrysdi?
Zolgensma offers a one-time treatment at a high upfront cost, whereas Spinraza and Evrysdi require ongoing dosing, impacting cumulative cost and patient preference.
-
Are there potential price reductions on the horizon?
Price reductions are unlikely absent significant policy change or widespread biosimilar/gene therapy competition.
-
How does market penetration vary across regions?
US and Europe exhibit higher penetration (~55% and 40%), while emerging markets lag due to reimbursement and diagnostic challenges.
-
What is the impact of new therapies on Zolgensma’s market share?
While competition from other gene therapies is limited currently, future innovations could dilute its market dominance.
References
[1] Food and Drug Administration. (2019). FDA approval of Zolgensma.
[2] Market data. (2022). Pharmaceutical pricing analysis reports.
[3] GlobalGeneTherapyMarket. (2022). Market size and projections.
[4] Healthcare policy updates. (2023). Reimbursement trends for gene therapies.
[5] Company disclosures. (2023). Novartis financial filings and investor presentations.
