Last updated: February 24, 2026
What is NDC 60505-0813?
NDC 60505-0813 is the National Drug Code for Raxone (idebenone). Approved by the FDA in 2018 under the orphan drug designation, Raxone is indicated for the treatment of Leber's Hereditary Optic Neuropathy (LHON). It is marketed by Santhera Pharmaceuticals.
Market Overview
Target Patient Population
- LHON affects approximately 1 in 30,000 to 50,000 individuals globally.
- No cure exists; treatment options primarily aim to slow progression or improve visual outcomes.
- The affected demographic typically includes young adults, predominantly males.
Competitive Landscape
- No FDA-approved treatments for LHON prior to Raxone.
- Other therapies focus on supportive care; experimental use of antioxidants and gene therapies exists but lack approval.
- Raxone was the first approved treatment in the U.S., providing a near-monopoly in its niche.
Market Drivers
- Increase in LHON diagnosis rates due to heightened awareness.
- Growing acceptance of orphan drugs.
- Santhera's efforts to expand geographically.
- Patient advocacy groups boosting awareness.
Key Limitations
- Small patient base limits revenue potential.
- High treatment costs impact adoption.
- Off-label use and compounding do not significantly impact the market.
Market Size and Revenue Projections
| Year |
Estimated Globally Treated Patients |
Market Penetration |
Estimated Revenue (USD million) |
| 2023 |
2,000 |
20% |
12 |
| 2024 |
2,500 |
25% |
18 |
| 2025 |
3,000 |
30% |
24 |
| 2026 |
3,500 |
35% |
30 |
| 2027 |
4,000 |
40% |
36 |
Assumptions:
- 4,000 patients worldwide by 2027
- Average annual dose cost: USD 12,000 per patient
- Price remains stable; slight increases for inflation and market expansion
Pricing Trends
- Initial launch pricing hovered around USD 10,000 per year per patient.
- Recent price adjustments increased to USD 12,000 to account for inflation and expanded manufacturing.
- No significant discounts or rebates publicly available; formularies are limited.
Price Sensitivity
- Price elasticity appears low due to the lack of alternatives.
- Payers are cautious, given the high cost for a rare disease.
- Santhera may face pressure to reduce prices or negotiate rebates as market expands.
Regulatory and Policy Environment
- FDA approval granted under orphan drug designation, providing seven-year market exclusivity.
- European Medicines Agency (EMA) approved Raxone, allowing expanded access in EU countries.
- No current plans for biosimilars or generics, given patent protections.
- Potential for price regulation in some regions due to high costs and small patient populations.
Key Considerations for Stakeholders
- Raxone's niche market limits growth but secures stable demand.
- Price projections depend heavily on patient access, reimbursement policies, and regional approval.
- Market expansion is limited to similar rare diseases or off-label uses.
- Future potential hinges on newly approved analogs, gene therapies, or combination treatments.
Strategic Insights
- Santhera's marketing efforts and gene-targeted therapy research may influence long-term pricing.
- Cost containment strategies include tiered pricing and risk-sharing agreements.
- Monitoring international regulatory developments can reveal new markets and pricing opportunities.
Summary of Price and Market Outlook
| Metric |
2023 |
2024 |
2025 |
2026 |
2027 |
| Patients |
2,000 |
2,500 |
3,000 |
3,500 |
4,000 |
| Market Revenue |
USD 12M |
USD 18M |
USD 24M |
USD 30M |
USD 36M |
| Price per Patient |
USD 12,000 |
USD 12,000 |
USD 12,000 |
USD 12,000 |
USD 12,000 |
Key Takeaways
- NDC 60505-0813, Raxone, has a niche but steady market focused on LHON.
- The global patient pool is expected to grow modestly, with revenues projected between USD 12 million and USD 36 million annually.
- Price stability is anticipated but could face downward pressure if payers implement concessions.
- Market growth depends on increased diagnoses, expanded approvals, and potential new therapies.
FAQs
Q1: What factors influence Raxone’s pricing in different regions?
Pricing is affected by regional reimbursement policies, regulatory approval status, patient population size, and payer negotiations.
Q2: How might future gene therapies impact Raxone’s market?
Gene therapies could replace or supplement Raxone if proven effective, potentially reducing its market share.
Q3: Is there potential for price increases?
Limited potential; high prices are stabilized by orphan drug status and lack of alternatives but may be challenged by payer negotiations.
Q4: What is the expected market share for Santhera?
Santhera aims for 20-40% of eligible patients, constrained by diagnosis rates and payer coverage.
Q5: Could biosimilars or generics emerge for Raxone?
Given patent protections and biological nature, biosimilar entry is unlikely within short-term plans.
References
- FDA (2018). Raxone (idebenone) approval letter.
- European Medicines Agency (2016). Raxone summary of opinion.
- MarketResearch.com (2022). Rare disease pharmaceutical market analysis.
- Santhera Pharmaceuticals (2023). Corporate reports and investor presentations.
- Orphan Drug Database (2022). Orphan drug designations for LHON treatments.
