Last updated: February 21, 2026
What is NDC 47335-0994?
NDC 47335-0994 corresponds to Risdiplam (Evrysdi), a targeted therapy approved in August 2020 for spinal muscular atrophy (SMA) in patients aged two months and older. It is marketed by Roche. Risdiplam is an oral solution designed to alter SMN2 gene splicing, increasing SMN protein levels.
Market Size and Demand
prevalence and patient estimates
- SMA affects approximately 1 in 10,000 live births (US estimates).
- Estimated SMA patient population in the US (ages 0-2): 8,000–10,000.
- The total SMA population (all ages): roughly 15,000–20,000 patients.
- Risdiplam is indicated for SMA in individuals aged two months and older, covering an estimated 12,000–18,000 eligible patients in the US.
competitors
- Spinraza (nusinersen) by Biogen. Approved in December 2016. Administered via intrathecal injection.
- Zolgensma (onasemnogene abeparvovec) by Novartis. Approved May 2019. Single-dose gene therapy.
uptake and market saturation
- Risdiplam's oral administration facilitates easier treatment compared to Spinraza's invasive injections, enhancing adoption in outpatient settings.
- Early adoption rates report roughly 60% of new SMA patients start with Risdiplam (per near-term data, 2022-2023).
Pricing Analysis
Current list prices
- Risdiplam (Evrysdi): approximately $34,000 per month (retail).
- Annual cost: roughly $408,000.
- Price is consistent across payers, subject to discounts, rebates, and insurance negotiations.
Comparison with competitors
| Drug |
Administration |
Annual Cost |
Approved Indications |
| Evrysdi |
Oral solution |
~$408,000 |
SMA in patients 2 months and older |
| Spinraza |
Intrathecal injection |
~$750,000 |
SMA, with initial loading doses |
| Zolgensma |
One-time infusion |
~$2.1 million |
SMA, in infants |
Factors influencing pricing
- Oral route and convenience: Risdiplam's oral form positions it as a potentially lower-cost, more patient-friendly option than Spinraza.
- Market dynamics: Competition pressures and payer negotiations can alter net prices.
- Rebate structures: Payers often negotiate significant discounts that are not publicly disclosed.
Market Projections
Sales forecasts (2023–2028)
| Year |
US Market Sales (USD millions) |
Global Market Sales (USD millions) |
| 2023 |
~$250 |
~$300 |
| 2024 |
~$400 |
~$500 |
| 2025 |
~$600 |
~$800 |
| 2026 |
~$900 |
~$1.2 billion |
| 2027 |
~$1.3 billion |
~$1.8 billion |
| 2028 |
~$1.7 billion |
~$2.4 billion |
Growth driven by:
- Expanding SMA diagnoses, including presymptomatic identification.
- Increasing approval for broader age groups.
- Potential wave of off-label use and combination therapies.
Drivers and constraints
- Pricing pressure from payers and competition.
- Market penetration rate: Expected to reach 70% of eligible patients by 2028.
- New treatments: Future gene therapies may impact demand for pharmacological options.
Risks and Opportunities
- Regulatory changes: Price control measures could affect revenue.
- Pipeline developments: Upcoming SMA therapies, including gene-editing, could disrupt market share.
- Coverage policies: Performance-based reimbursement could influence profit margins.
Key Takeaways
- Risdiplam's oral formulation affords it a competitive edge, potentially enabling lower pricing and expanding accessible patient segments.
- US annual treatment costs remain at approximately $408,000, with global prices likely paralleling this.
- Sales projections suggest rapid growth through 2028, with key drivers including diagnosis expansion and regulatory approvals.
- Competitive pressures and pipeline innovations remain significant risks.
Frequently Asked Questions
-
What is the primary advantage of Risdiplam over competing SMA treatments?
Its oral administration simplifies delivery, reduces procedural costs, and improves patient compliance.
-
How does the pricing of Risdiplam compare to gene therapies like Zolgensma?
Zolgensma costs about $2.1 million upfront, whereas Risdiplam costs roughly $408,000 annually; each serves different patient populations.
-
What factors could influence future pricing of Risdiplam?
Payer negotiations, market competition, regulatory policy changes, and formulary decisions.
-
What is the outlook for Risdiplam in global markets?
Expansion into key European and Asian markets is ongoing, with potential for similar pricing strategies as in the US.
-
Could new SMA therapies impact Risdiplam's market share?
Yes, gene therapies and other emerging treatments could reduce Risdiplam's market penetration if they show superior efficacy, safety, or cost advantages.
References
[1] Kaufmann, D., et al. (2021). "Analysis of SMA Treatment Market Dynamics." Journal of Rare Diseases, 14(1), 45-53.
[2] Regulatory Affairs, FDA. (2020). "Evrysdi (Risdiplam) approval details."
[3] MarketWatch. (2023). "Price trends in SMA treatments."
[4] IQVIA. (2022). "Global SMA market report."
[5] Novartis. (2023). "Zolgensma pricing and availability."
