Last updated: February 15, 2026
What is the Drug Represented by NDC 70512-0860?
The National Drug Code (NDC) 70512-0860 corresponds to Eteplirsen (Exondys 51 300 mg/2.5 mL, intravenous), developed by Sarepta Therapeutics. It is approved by the FDA for the treatment of Duchenne Muscular Dystrophy (DMD) in patients with a confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping.
What is the Current Market Size and Demand?
Patient Population:
- The estimated DMD patient population in the U.S. is approximately 15,000.
- Of these, about 13% (roughly 2,000) have mutations treatable with exon 51 skipping, the indication for eteplirsen.
- The global clinic population is higher, but U.S. demand significantly influences pricing and market projections, especially due to reimbursement policies.
Market Dynamics:
- Niche market with limited patient pool.
- An unmet medical need exists, as eteplirsen is among few exon-skipping therapies approved.
Competitive Landscape:
- Other exon-skipping agents include Vyondys 53 (golodirsen) and Viltolarsen (Viltepso), both FDA-approved for different exon skipping mutations.
- No direct competition for exon 51 in the current market; off-label treatments include corticosteroids and supportive care, which do not address mutation-specific therapy.
What is the Pricing of NDC 70512-0860?
Current Price Points:
- The list price for eteplirsen has been approximately $375,000 per year per patient (average wholesale price basis, as of 2022).
- Variants with different dosing schedules or variations in packaging may influence actual costs.
Reimbursement Environment:
- Medicare, Medicaid, and private insurers typically reimburse near list prices after negotiations and patient assistance programs.
- Insurance coverage issues could influence actual payer costs and patient access rates.
What Are the Price Trends and Projections?
| Year |
Approximate Price (per patient/year) |
Notes |
| 2022 |
$375,000 |
Current list price. |
| 2023 |
$375,000 |
Stable, barring regulatory or patent changes. |
| 2024 |
$370,000 - $380,000 |
Slight adjustments expected. |
| 2025 |
$365,000 - $385,000 |
Slight inflation-adjusted variations. |
| 2026 |
$360,000 - $390,000 |
Possible price erosion or premium increase based on competition and outcomes data. |
What Factors Could Impact Market and Price Trajectory?
- Regulatory Changes: FDA decisions impacting approval, pricing, or additional indications.
- Competitive Innovations: New exon-skipping therapies or gene editing approaches could disrupt demand.
- Reimbursement Policies: Payer negotiations, formulary inclusion, and patient assistance programs influence net pricing.
- Market Penetration: Adoption rates depend on physician acceptance and patient accessibility.
- Clinical Data: Demonstrated long-term efficacy and safety could justify premium pricing versus newer or generic options.
What is the Outlook Beyond 2025?
- Price stabilization or slight decrease likely unless significant improvements in efficacy or expanded indications occur.
- Demand may grow as awareness increases and older patients seek treatment.
- Entry of competitors and potential biosimilars could drive prices downward.
Key Takeaways
- The current annual price for NDC 70512-0860 is approximately $375,000 per patient.
- The target market primarily comprises about 2,000 U.S. patients with exon 51 mutations.
- Price projections indicate slight inflation or stability, with potential downward pressure from emerging therapies and market competition.
- Market size remains limited but critical for Sarepta and players in rare disease therapeutics.
- Reimbursement policies and clinical outcomes will shape future pricing and market penetration.
FAQs
What is the primary indication for NDC 70512-0860?
Treatment of Duchenne Muscular Dystrophy in patients with confirmed exon 51 skipping mutation.
What are the main competitors?
Vyondys 53 (golodirsen) and Viltolarsen (Viltepso), approved for other exon skipping mutations.
How does the pricing compare to other rare disease drugs?
Eteplirsen’s $375,000 per year is aligned with other high-cost rare disease treatments, which often range from $250,000 to over $500,000 annually.
What are the main factors influencing future prices?
Regulatory changes, clinical efficacy data, market competition, reimbursement policies, and patient access.
What is the potential global market for eteplirsen?
The global market expands roughly three to four times the U.S. size, with similar mutation prevalence, but pricing and reimbursement vary significantly across countries.
References
[1] FDA Approval. (2016). Eteplirsen for Duchenne Muscular Dystrophy. FDA.gov.
[2] Sarepta Therapeutics. (2022). Product Pricing and Market Access Strategies.
[3] IQVIA. (2022). Global Orphan Drug Market Trends.
[4] Evaluated Pricing Data from Various Payer Sources.
[5] Drug Channels Institute. (2023). Pricing Trends for Rare Disease Drugs.
Note: All price figures are estimates based on publicly available data as of early 2023 and may vary with negotiations and regional pricing policies.
