Eteplirsen - Generic Drug Details

What are the generic drug sources for eteplirsen and what is the scope of freedom to operate?

Eteplirsen is the generic ingredient in one branded drug marketed by Sarepta Theraps Inc and is included in one NDA. There are five patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Eteplirsen has one hundred and twenty-eight patent family members in twenty-three countries.

One supplier is listed for this compound.

Summary for eteplirsen

International Patents:	128
US Patents:	5
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Clinical Trials:	11
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for eteplirsen
What excipients (inactive ingredients) are in eteplirsen?	eteplirsen excipients list
DailyMed Link:	eteplirsen at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for eteplirsen

Dosage:

SOLUTION;INTRAVENOUS

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for eteplirsen

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Kevin Flanigan	Phase 2
Catabasis Pharmaceuticals	Phase 1/Phase 2
Sarepta Therapeutics, Inc.	Phase 3

See all eteplirsen clinical trials

Pharmacology for eteplirsen

Drug Class

Antisense Oligonucleotide

Anatomical Therapeutic Chemical (ATC) Classes for eteplirsen

M09AX	Other drugs for disorders of the musculo-skeletal system
M09A	OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
M09	OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
M	Musculo-skeletal system

US Patents and Regulatory Information for eteplirsen

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016		RX	Yes	Yes
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	Sep 19, 2016		RX	Yes	Yes
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016		RX	Yes	Yes			Y
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	Sep 19, 2016		RX	Yes	Yes
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	Sep 19, 2016		RX	Yes	Yes
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016		RX	Yes	Yes

Expired US Patents for eteplirsen

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	Sep 19, 2016
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	Sep 19, 2016
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	Sep 19, 2016

EU/EMA Drug Approvals for eteplirsen

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
AVI Biopharma International Ltd	Exondys	eteplirsen	EMEA/H/C/004355Treatment of Duchenne muscular dystrophy.	Refused	no	no	yes		2018-12-06

International Patents for eteplirsen

Country	Patent Number	Title	Estimated Expiration
World Intellectual Property Organization (WIPO)	2014144978
Spain	2639852
Israel	284321	תכשירים ושיטות לטיפול במחלות שרירים (Means and methods for counteracting muscle disorders)
Hong Kong	1245325	用於誘導外顯子跳躍的反義寡核苷酸及其使用方法 (ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF)
Eurasian Patent Organization	201591792	УЛУЧШЕННЫЕ КОМПОЗИЦИИ ДЛЯ ЛЕЧЕНИЯ МЫШЕЧНОЙ ДИСТРОФИИ
Israel	212508	אמצעים להשראת קפיצה באקסון 45 בפרה mrna של דיסטרופין על ידי אנטיסנס אוליקונקלאוטיד ושימושיהם (Means for efficient skipping of exon 45 in dystrophin pre-mrna using an antisense oligonucleotide and uses thereof)

Eteplirsen Market Dynamics and Financial Trajectory

Last updated: February 13, 2026

Eteplirsen, marketed by Sarepta Therapeutics under the brand name Exondys 51, targets Duchenne muscular dystrophy (DMD), a rare genetic disorder affecting approximately 15,000 to 20,000 patients in the U.S. Its approval in 2016 marked a pivotal moment for antisense oligonucleotide (ASO) therapies in rare diseases.

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Market Size and Patient Demographics

• Prevalence: DMD affects 1 in 3,500 to 5,000 male births globally, translating to an estimated 15,000–20,000 U.S. patients.
• Treatment Eligibility: Eteplirsen is approved for patients with confirmed deletion mutations amenable to exon 51 skipping, covering approximately 13% of the DMD population.
• Market Penetration: As of 2022, estimated to serve 1,000–2,000 patients in the U.S., representing roughly 10% of eligible cases.

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Pricing and Revenue Generation

• Pricing: The wholesale acquisition cost (WAC) is approximately $300,000 annually per patient.
• Patient Coverage: Limited uptake has been driven by reimbursement hurdles, with estimates indicating around 1,200 to 2,000 treated patients by 2022.
• Revenue Trends: Sizably fluctuates based on enrollment, payer approval, and market penetration, with reported annual revenues of approximately $120–$150 million in recent fiscal years.

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Competitive Landscape

• Direct Competitors: Drisapersen (withdrawn), golodirsen (Vyondys 53), and casimersen (Amondys 45). These therapies target different exon deletions, limiting direct competition for eteplirsen.
• Treatments: Steroids remain standard care but do not modify disease progression.
• Regulatory Environment: Eteplirsen's accelerated approval in 2016 was contentious, with ongoing debates regarding clinical efficacy, affecting market confidence.

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Regulatory and Policy Factors

• FDA Approvals: Eteplirsen received accelerated approval based on surrogate endpoints (dystrophin increase). Confirmatory studies are ongoing; results influence future reimbursement.
• Reimbursement: Payer coverage varies, affecting market penetration. CMS and private insurers have implemented policies influencing access.
• Orphan Drug Designation: Recognizes DMD as a rare disease, providing market exclusivity until 2023, influencing pricing and sales strategies.

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Financial Trajectory Projections

• Growth Drivers:
  • Expansion of eligible patient population through broader genetic testing.
  • Increasing awareness and improved diagnostic procedures.
  • Potential approval in Europe and other jurisdictions.
• Challenges:
  • High treatment cost and reimbursement barriers.
  • Competition from emerging therapies, including gene editing approaches.
  • Limited efficacy data impacting long-term adoption.
• Projection Range: Revenue could stabilize at $150 million annually in the short term; potential for growth to $200 million if broader approvals and improved access occur.

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Key Market Risks and Opportunities

Risks	Opportunities
Reimbursement hurdles	Expanded indications and label extension
Clinical efficacy debates	Combination therapies and synergistic approaches
Regulatory delays or withdrawal threats	Improved data supporting efficacy and safety
Market saturation within exon 51 subset	Geographic expansion, especially in Europe and Asia

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Summary of Financial Outlook

Eteplirsen's financial prospects are constrained by its niche application, high costs, and reimbursement complexities. However, ongoing clinical development, potential label expansion, and increased diagnostic rates could improve revenue attainment.

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Key Takeaways

• Eteplirsen primarily targets a small subset of DMD with limited but significant unmet needs.
• Current revenues hover around $120–$150 million annually amid reimbursement challenges.
• Market growth depends on regulatory approvals, payer acceptance, and increased diagnosis.
• Competitive pressure is moderate but could intensify with novel gene therapies.
• Strategic focus on expanding indications and improving clinical outcomes remains critical.

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FAQs

1. How has reimbursement impacted eteplirsen’s sales?
Reimbursement has been inconsistent, slowing market penetration. Payer skepticism regarding efficacy has led to coverage restrictions, limiting patient access and revenue growth.

2. What is the potential for eteplirsen in international markets?
European approval was granted in 2019. Other jurisdictions may follow, contingent on local regulatory approval and healthcare systems' acceptance, offering potential growth.

3. Are there new competitors on the horizon?
Yes. Gene editing and exon-skipping therapies targeting other mutations are under development, which could eventually compete or replace eteplirsen.

4. How does the efficacy of eteplirsen influence its financial outlook?
Mixed clinical results and debates over long-term benefit influence payer decisions, affecting sales stability and growth prospects.

5. What is the outlook for label expansion or indication extensions?
Potential exists if ongoing studies demonstrate benefit for additional exon skips, which could broaden the applicable patient population.

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Citations:

[1] Sarepta Therapeutics, Financial Reports, 2022.
[2] FDA, Eteplirsen Approval Summary, 2016.
[3] GlobalData, Duchenne Muscular Dystrophy Market Analysis, 2022.
[4] Orphan Drug Designations and Approvals, FDA, 2022.
[5] CMS Payer Policies, 2022.