Last updated: February 25, 2026
What Is the Drug NDC 13107-0070?
The National Drug Code (NDC) 13107-0070 corresponds to Eteplirsen (Exondys 51). It is an approved treatment for Duchenne Muscular Dystrophy (DMD) caused by specific genetic mutations that affect exon 51 splicing. Approved by the FDA in 2016, it is marketed by Sarepta Therapeutics.
Market Size and Demand
Patient Population
- DMD affects approximately 1 in 3,500 to 5,000 male births.
- Estimated U.S. prevalence: 10,000–15,000 patients, with about 13% eligible for exon 51 skipping therapy.
- On-label eligible patients: approximately 1,300–2,000 in the U.S.
Competitive Landscape
- Only one approved therapy targeting exon 51 skipping: Eteplirsen.
- Competing or pipeline therapies include golodirsen and casimersen.
Market Penetration
- Estimated initial penetration: 10-15% of eligible patients in the first year.
- Penetration growth over five years: up to 50% with evolving approval and reimbursement strategies.
Pricing and Reimbursement
Current List Price
- The list price of Eteplirsen stands at approximately $375,000 per year per patient (per 48-week course).
Net Realized Price
- After discounts, rebates, and insurance negotiations, the net price is estimated to be $250,000–$300,000 annually.
Price Trends
- Price has remained stable since initial launch, with minor annual increases (~3-5%) in line with inflation and cost adjustments.
Revenue Projections
| Year |
Estimated Patients |
Market Penetration |
Total Revenue (USD) |
| 2023 |
1,300 |
10% |
~$325 million |
| 2024 |
1,700 |
15% |
~$510 million |
| 2025 |
2,000 |
20% |
~$600 million |
| 2026 |
2,500 |
30% |
~$750 million |
| 2027 |
3,000 |
50% |
~$900 million |
Assumptions: Price per patient remains stable. Penetration increases as awareness and approval expand.
Regulatory and Pricing Policy Influence
- External pressures to reduce prices include payer negotiations and patient access programs.
- Pricing strategies may shift as biosimilars or alternative exon skipping therapies develop.
- Expansion into additional markets (e.g., Europe, Japan) could influence revenues and pricing.
Competitive Dynamics and Future Outlook
- As pipeline therapies enter, market share could shift.
- Patent expirations or biosimilar entries, if applicable, could impact pricing.
- Advances in gene therapies may alter the landscape after 2030.
Key Market Drivers
- Expansion of approved indications.
- Increased diagnosis rates.
- Reimbursement access improvements.
- Broader acceptance among clinicians.
Risks and Challenges
- Price sensitivity among payers.
- Patient adherence and access barriers.
- Competition from emerging therapies.
Summary
Eteplirsen (NDC 13107-0070) maintains a high list price with stable demand among exon 51 skipping-eligible patients. Revenue projections range from approximately $325 million in 2023 to nearly $900 million by 2027, contingent on market penetration and reimbursement strategies.
Key Takeaways
- The market for NDC 13107-0070 centers on a small patient cohort with stable, premium pricing, and significant growth potential.
- Demand depends on diagnosis rates and reimbursement access, with revenues likely to increase moderately over five years.
- Competitive pipeline developments and policy pressures pose risks to sustained pricing and market share.
FAQs
1. How does the price of Eteplirsen compare to other Duchenne therapies?
Eteplirsen’s list price is roughly comparable to other high-cost DMD treatments. For example, Emflaza (deflazacort) costs around $25,000 annually, but it is a corticosteroid, not exon-specific. Other exon-skipping drugs like casimersen are priced similarly; exact comparisons depend on negotiated net prices.
2. What factors could cause the price to decline?
Introduction of biosimilars, new pipeline therapies, or increased payer pressure could lead to price reductions. Cost-containment policies and broader reimbursement negotiations may also influence pricing.
3. How do international markets impact overall revenue?
Europe, Japan, and other regions are potential growth markets. Price points in these regions are generally lower, and market entry depends on regulatory approvals and pricing negotiations.
4. What is the primary driver of market growth for NDC 13107-0070?
The most significant driver is the increasing diagnosis rate of eligible DMD patients and expanded reimbursement access, boosting demand over the next five years.
5. Are there upcoming regulatory changes that could impact this drug?
Yes. Revisions in healthcare policy, reimbursement criteria, and approval pathways for rare disease drugs could influence market access and pricing strategies.
References
- Sarepta Therapeutics. (2022). Exondys 51 (Eteplirsen) Prescribing Information. Retrieved from https://www.sarepta.com.
- IQVIA. (2022). U.S. Market Data for Rare Disease Treatments.
- FDA. (2016). Approval of Eteplirsen for Duchenne Muscular Dystrophy. Retrieved from https://www.fda.gov.
- MedPage Today. (2022). Pricing and Reimbursement of DMD Therapies.
- GlobalData. (2023). Orphan Drug Market Forecast – 2023-2028.
Note: Market figures and projections are estimates based on available data as of Q1 2023 and may vary with actual market developments.
