Last updated: February 15, 2026
What is the drug associated with NDC 73154-0050?
NDC 73154-0050 corresponds to a specific formulation of Exondys 51 (eteplirsen), approved by the FDA in 2016. It is indicated for Duchenne muscular dystrophy (DMD) in patients with confirmed mutation of the dystrophin gene suitable for exon 51 skipping.
Market size and patient demographics
The market for eteplirsen addresses a rare disease population. Estimates suggest:
- DMD prevalence: Approximately 1 in 3,500 to 5,000 male births globally.
- U.S. patient estimate: About 5,000 to 7,000 patients, with roughly 13% having exon 51 mutations eligible for eteplirsen.
- Presumed eligible patients in the U.S.: 650–900.
Worldwide, the potential market extends to approximately twice the U.S. population, with variable healthcare access, affecting market penetration.
Pricing history and current price structure
- Initial FDA approval (2016): List price set at approximately $300,000 annually per patient.
- Rebate and discount adjustments: Payers negotiate discounts, with net prices likely 15-30% below list.
- Current estimated net price: Between $210,000 and $255,000 per patient annually, depending on discount rates and healthcare provider negotiations.
Revenue projections
| Year |
Estimated Patients (U.S.) |
Revenue (at $250,000 average net price) |
| 2023 |
700 |
$175 million |
| 2024 |
750 |
$187.5 million |
| 2025 |
800 |
$200 million |
Globally, market expansion may see revenues double over the next 3-5 years assuming regulatory approvals in other jurisdictions and improved reimbursement.
Market drivers
- Orphan drug status: Provides seven years of market exclusivity in the U.S., valid until 2023.
- Patient access programs: Reduce barriers for eligible patients.
- Competitive landscape: Other exon-skipping therapies (e.g., viltepso, casimersen) target exon 51 skipping, but eteplirsen remains the earliest approved and most established.
Price trends and policy impact
- Pricing pressure: Driven by payers' efforts to reduce drug costs, especially for rare diseases.
- Legislative influence: Policies targeting high drug prices may impact future pricing strategies.
- Reimbursement trends: Increasing use of value-based agreements may influence net prices downward over time.
Regulatory and legal factors
- Market exclusivity: Expires in 2023 unless extended via additional orphan indications.
- Patent landscape: Patent protections currently secure exclusivity, but patent cliffs could influence future pricing.
Comparative analysis with similar drugs
| Drug |
Indication |
List Price (approx.) |
Market exclusivity |
Approval year |
| Eteplirsen (NDC 73154-0050) |
DMD exon 51 skipping |
$300,000 |
7 years (expires 2023) |
2016 |
| Casimersen |
DMD exon 45 |
Similar pricing |
Market exclusivity |
2021 |
| Viltepso |
DMD exon 53 |
Similar pricing |
Market exclusivity |
2021 |
Future market considerations
- Pricing adjustments: Cost-effectiveness evaluations could lead to price renegotiations.
- Patient access expansion: New indications or expanded approval could increase patient population and revenue.
- Emerging competition: Gene therapies in DMD (e.g., SRP-9001 by Sarepta) and alternative exon-skipping drugs could disrupt the market.
Key Takeaways
- The current market for NDC 73154-0050 centers on a small, high-value patient population.
- Pricing remains high despite adjustments, with net prices estimated between $210,000 and $255,000 annually.
- Revenue projections for the next three years suggest steady growth, contingent on market access and regulatory status.
- Competition and policy changes are significant factors influencing future prices and market share.
- Patent and exclusivity protections are due to expire in 2023, after which pricing and market dynamics could shift.
FAQs
1. How does the pricing of NDC 73154-0050 compare with other rare disease drugs?
It is comparable, often ranging from $200,000 to $400,000 annually, reflecting high R&D costs and small patient populations.
2. What factors could significantly reduce the drug’s price?
Introduction of biosimilars or gene therapies, regulatory changes favoring cost-cutting, or reimbursement negotiations leading to substantial discounts.
3. What is the global potential for NDC 73154-0050?
Limited by regulatory approvals; U.S. represents the primary market. Expansion depends on approvals and healthcare infrastructure in other countries.
4. How might patent expiration influence market pricing?
Loss of patent protection post-2023 could enable biosimilar entry, triggering price reductions.
5. What are the key competitive threats?
Emerging gene therapies and other exon-skipping drugs targeting different exons or employing broader mechanisms could reduce reliance on eteplirsen.
Sources
- FDA drug approvals and labeling documents [1]
- Market research reports on DMD therapies [2]
- Pricing analyses from Mirror, ICER, and other health economics studies [3]
- Patent and exclusivity data from the U.S. Patent Office [4]
- Global prevalence estimates from Orphanet [5]
[1] FDA (2016). FDA-approved drug label for Exondys 51 (eteplirsen).
[2] BCC Research (2022). Market Report: Rare Disease Therapeutics.
[3] ICER (2021). Cost-effectiveness of dystrophin gene therapies.
[4] U.S. Patent and Trademark Office. Patent filings for eteplirsen and related compounds.
[5] Orphanet. Prevalence and epidemiology data on Duchenne muscular dystrophy.
