Last updated: February 27, 2026
What is the drug with NDC 46122-0628?
The drug identified by NDC 46122-0628 is Risdiplam (Evrysdi). It is an oral SMN2 splicing modifier approved by the FDA in August 2020 for treating spinal muscular atrophy (SMA) in both pediatric and adult patients. It is developed by Roche.
Market Overview
Indications and Patient Population
Risdiplam targets SMA, a rare neuromuscular disorder characterized by loss of motor neurons, leading to muscle weakness. The drug’s approved indication covers:
- Infants
- Children
- Adults with SMA
The total addressable market (TAM) depends on the prevalence of SMA, which is approximately 1 in 10,000 live births globally, translating to an estimated 15,000-20,000 patients in the U.S. alone.
Competitive Landscape
Major competitors include:
- Spinraza (nusinersen) by Biogen
- Zolgensma (onasemnogene abeparvovec) by Novartis
- Evrysdi (risdiplam) by Roche
Both Spinraza and Zolgensma have existing market share. Spinraza launched in 2016, leading in revenue until the approval of risdiplam.
Market Penetration and Adoption
By mid-2022, Evrysdi had obtained a significant market share, especially among newly diagnosed SMA patients due to its oral administration.
The global SMA drug market was valued at approximately USD 1.2 billion in 2021. Projected compound annual growth rate (CAGR) for the SMA drug market is around 8-10% through 2027.
Price Analysis
Current Pricing
- Evrysdi (Risdiplam):
- U.S. list price: approximately USD 340,000 - USD 360,000 annually, depending on dosing and patient weight.
- Spinraza:
- Initial dose: USD 750,000, with follow-up doses around USD 125,000 per injection.
- Zolgensma:
- One-time infusion priced at approximately USD 2.125 million.
Pricing Strategy and Reimbursement
Roche’s pricing reflects:
- The convenience of oral administration over injectable formulations.
- Pricing alignment with the existing SMA market.
- Reimbursement status varies by country and insurance coverage; favorable in the U.S. due to coverage policies under Medicare and Medicaid.
Price Trends and Projections
Given market competition and pricing pressures:
- The USD 340,000 - USD 360,000 range is expected to remain stable through 2024.
- As biosimilar or generic options emerge, prices could decline by 5-10% annually post-2025.
- Increased adoption will predominantly be driven by lower-cost access and expanded indications, such as newer patient segments.
Revenue Projection Strategy for Roche
Based on market size, reimbursement rates, and competitive dynamics:
| Year |
Estimated Patients (U.S.) |
Market Penetration |
Approximate Revenue (USD millions) |
| 2022 |
3,000 |
20% |
102 |
| 2023 |
4,500 |
30% |
153 |
| 2024 |
6,000 |
40% |
216 |
| 2025 |
7,500 |
50% |
270 |
Assumptions: steady market growth, gradual increase in patient access, stable pricing.
Market Risks and Opportunities
Risks
- Entry of biosimilars or generics leading to price erosion.
- Reimbursement challenges and payer negotiations.
- Uncertainty from emerging therapies or gene editing technologies.
Opportunities
- Broader label expansion for neonatal and adult SMA.
- Uptake in off-label indications, such as multiple sclerosis.
- Pricing flexibility in emerging markets.
Key Takeaways
- NDC 46122-0628 (Risdiplam) has solidified its position as a key oral therapy in SMA treatment.
- Launch prices stand around USD 340,000 – USD 360,000 annually.
- The total addressable market in the U.S. is approximately 15,000-20,000 patients, with global revenues expected to grow at 8-10% CAGR through 2027.
- Revenue growth is constrained by competitive pressure and potential biosimilar entry but supported by expanding indications.
- Future pricing may decrease by up to 10% annually after 2025 as biosimilars and new therapies increase market competition.
FAQs
What is the expected launch price of Risdiplam in new markets?
Pricing will reflect local healthcare economics, but it is expected to be similar proportionally to U.S. prices, adjusted for market size and reimbursement policies.
How does Risdiplam's pricing compare to Spinraza and Zolgensma?
Risdiplam’s annual cost (~USD 340,000–USD 360,000) is lower than Zolgensma's one-time USD 2.125 million but similar to Spinraza’s ongoing treatment cost after initial dosing.
What are the primary factors influencing Risdiplam pricing?
Market competition, treatment convenience, manufacturing costs, payer negotiations, and regulatory reimbursement policies.
How will biosimilars impact Risdiplam’s market share?
Introduction of biosimilars could reduce prices by 5-10% annually post-2025, pressuring Roche’s revenue growth.
Are there off-label uses for Risdiplam?
Currently, approved for SMA. Off-label use is limited; no significant evidence supports expansion beyond approved indications at this time.
References
[1] IQVIA. (2022). Global SMA Market and Therapy Overview.
[2] Food and Drug Administration (FDA). (2020). FDA Approves Evrysdi for Treatment of Spinal Muscular Atrophy.
[3] EvaluatePharma. (2022). Biopharma Market Analysis, SMA Segment.
[4] Roche. (2022). Evrysdi (Risdiplam) Corporate Data and Pricing Details.
