ETEPLIRSEN - Generic Drug Details

What are the generic sources for eteplirsen and what is the scope of patent protection?

Eteplirsen is the generic ingredient in one branded drug marketed by Sarepta Theraps Inc and is included in one NDA. There are eight 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:	8
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

Constraining patent/regulatory exclusivity:

RE48468

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	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	206488-001	Sep 19, 2016		RX	Yes	Yes	10,337,003
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	Sep 19, 2016		RX	Yes	Yes	10,364,431
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	Sep 19, 2016		RX	Yes	Yes	RE48468
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	Sep 19, 2016		RX	Yes	Yes	9,018,368		Y	Y
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	Sep 19, 2016		RX	Yes	Yes	RE47751
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016		RX	Yes	Yes	RE47751
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016		RX	Yes	Yes	10,337,003

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	9,018,368
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016	RE47751
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	Sep 19, 2016	9,416,361
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	Sep 19, 2016	10,533,174
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016	10,533,174
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016	9,416,361
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016	8,486,907

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
Portugal	2203173
European Patent Office	2607484	Procédé et moyens d’induction du saut de l’exon 45 dans l’ARN pré-messager du gène de la dystrophie musculaire de Duchenne (Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA)
China	113633787	改进的用于治疗肌营养不良的组合物 (Improved compositions for treating muscular dystrophy)
Eurasian Patent Organization	201591792	УЛУЧШЕННЫЕ КОМПОЗИЦИИ ДЛЯ ЛЕЧЕНИЯ МЫШЕЧНОЙ ДИСТРОФИИ
China	105307723	Improved compositions for treating muscular dystrophy
European Patent Office	2344637	PROCÉDÉS ET MOYENS D INDUCTION DU SAUT DE L EXON 45 DANS L ARN PRÉ-MESSAGER DU GÈNE DE LA DYSTROPHIE MUSCULAIRE DE DUCHENNE (METHODS AND MEANS FOR EFFICIENT SKIPPING OF EXON 45 IN DUCHENNE MUSCULAR DYSTROPHY PRE-MRNA)
Canada	2906812	COMPOSITIONS AMELIOREES POUR LE TRAITEMENT DE LA DYSTROPHIE MUSCULAIRE (IMPROVED COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY)

Market Dynamics and Financial Trajectory for Eteplirsen

Last updated: July 28, 2025

Introduction

Eteplirsen, marketed as EXONDYS 51, is a pioneering therapeutic developed by Sarepta Therapeutics for treating Duchenne Muscular Dystrophy (DMD), a rare genetic disorder characterized by progressive muscle degeneration. As one of the first exon-skipping therapies approved specifically for DMD, eteplirsen’s market presence illustrates the complex interplay of scientific innovation, regulatory landscape, and commercial viability in the orphan drug space.

This analysis explores the dynamic factors influencing eteplirsen’s market trajectory, revenue potential, and competitive landscape, providing stakeholders with actionable insights into its financial outlook.

Market Landscape for Duchenne Muscular Dystrophy

Prevalence and Patient Demographics

DMD primarily affects boys, with an estimated prevalence of approximately 1 in 3,500 to 5,000 male births globally. The rarity classifies it as a rare disease, or orphan condition, which influences regulatory incentives and market structure.

Unmet Medical Needs

No curative treatments previously existed for DMD. Management has focused on corticosteroids and supportive care, emphasizing the high unmet need that eteplirsen sought to address upon approval in 2016. The target demographic represents a relatively small patient population but one with significant medical urgency.

Regulatory Environment and Approvals

Eteplirsen's approval by the U.S. FDA in 2016 marked a milestone as the first exon 51-skipping therapy for DMD. It received accelerated approval, contingent upon post-marketing confirmatory trials, reflecting regulatory eagerness to facilitate access to promising orphan drugs.

Other jurisdictions, such as the European Union, have also granted conditional or full approvals, though with more cautious stances, influencing global sales patterns.

Market Dynamics Influencing Eteplirsen’s Trajectory

1. Scientific and Clinical Efficacy Perceptions

Eteplirsen’s clinical efficacy remains a point of debate within the medical community. While it demonstrated an increase in dystrophin protein levels, critics questioned the clinical significance and long-term benefits, impacting physician prescribing patterns and insurance coverage.

This skepticism affects market penetration, with off-label use limited, and influences the competitive landscape as newer therapies emerge.

2. Competitive Innovations and Emerging Therapies

Since eteplirsen’s approval, several other exon-skipping agents have entered or are advancing through clinical trials, including vyondys 53 (golodirsen) and casimersen. Additionally, gene editing approaches such as CRISPR-based therapies promise potential breakthroughs, threatening eteplirsen’s market share.

The competitive pipeline and potential approvals are critical determinants of eteplirsen’s future sales, necessitating ongoing innovation and differentiation strategies.

3. Reimbursement and Pricing Strategies

Pricing for eteplirsen has been contentious, initially set at approximately $300,000 annually per patient, aligning with other orphan drugs. Payers and health authorities scrutinize cost-effectiveness, especially with mixed clinical efficacy data.

Reimbursement policies significantly impact patient access and revenue streams, with payer resistance leading to coverage restrictions in certain markets. Market access negotiations and pricing adjustments are vital to sustain financial performance.

4. Patient Advocacy and Market Penetration

Strong advocacy groups bolster awareness and drive demand, but their influence is moderated by clinical efficacy debates. The limited diagnosed patient pool constrains volume growth, while the high cost per patient amplifies revenue potential but also regulatory and market entry challenges.

5. Regulatory and Policy Developments

Future regulatory decisions, including potential reversals of accelerated approvals or additional post-market data requirements, could alter eteplirsen’s market prospects. Policy shifts favoring stricter efficacy benchmarks could restrict sales growth.

Financial Trajectory and Revenue Outlook

Current Revenue Performance

Eteplirsen’s initial sales post-approval hovered around $50-100 million annually, reflecting limited patient adoption due to clinical uncertainties and reimbursement hurdles. Sarepta’s strategic focus on expanding indications and improving clinical data aims to bolster revenue streams.

Projected Growth Factors

Factors influencing future revenue include:

• Market Penetration: Increasing diagnosis rates and expanded access could raise patient numbers. Current estimates suggest approximately 10-15% of eligible patients receive eteplirsen, with room for growth through awareness and reimbursement expansion.

• Pipeline and Label Expansion: The development of casimersen and approvable exon 45-skipping therapies widen the treatment landscape, potentially capturing a larger patient subset and fostering market competition.

• Pricing Adjustments: Negotiated price reductions or value-based pricing agreements could stabilize revenue streams amid payer pressures.

• Long-term Efficacy Data: Demonstrable clinical benefits over extended periods can solidify payer support and promote broader use, catalyzing revenue growth.

Long-term Financial Outlook

Analysts project eteplirsen’s peak sales could reach $200-300 million annually within 5 years, contingent on improved efficacy evidence, expanded patient access, and favorable regulatory policies. However, the advent of gene editing and next-generation therapies could temper growth, establishing eteplirsen as a niche or foundational therapy rather than a dominant player.

Market Risk and Opportunities

Risks

• Efficacy and Regulatory Scrutiny: Ongoing validation concerns could impair market confidence.
• Competitive Pressures: Embrace of novel therapies may render eteplirsen less attractive.
• Pricing and Reimbursement: Payer resistance and cost containment policies limit revenue expansion.

Opportunities

• Label Expansion: Approvals for additional exon-skipping indications can broaden the target population.
• Combination Treatments: Integration with other therapies might enhance clinical outcomes.
• Patient Engagement: Advocacy-driven awareness campaigns could improve diagnosis and treatment adoption.

Conclusion: The Road Ahead

Eteplirsen’s market trajectory is shaped by scientific credibility, competitive landscape evolution, regulatory frameworks, and pricing strategies. While the therapy pioneered a new treatment paradigm for DMD, ongoing challenges necessitate strategic agility. Stakeholders should monitor clinical data developments, policy shifts, and emerging technologies to optimize investment and commercialization strategies.

Key Takeaways

• Eteplirsen remains a pioneering but contested therapy within a niche, with projected peak sales around $200-300 million, subject to clinical validation and payer acceptance.
• The evolving landscape of exon-skipping therapies and gene editing technologies poses both competitive threats and collaboration opportunities.
• Effective market access strategies, including demonstrating long-term clinical benefits and managing pricing negotiations, are critical for sustained revenue.
• Expanding indications through regulatory approvals could significantly increase the addressable patient population.
• Continuous post-market studies and real-world evidence will be pivotal in maintaining regulatory support and payer confidence.

FAQs

1. What is the current status of eteplirsen’s clinical efficacy?
While eteplirsen increases dystrophin levels, its long-term clinical benefits have been debated. Recent trials aim to validate efficacy further and influence payer and physician confidence.

2. How does eteplirsen compare with newer DMD therapies?
Emerging therapies, including gene editing and next-generation exon-skipping agents, may offer more comprehensive efficacy, positioning eteplirsen as a foundational therapy rather than a future leader.

3. What is the primary market challenge for eteplirsen?
Reimbursement and clinical efficacy skepticism are major hurdles, affecting patient access and sales growth.

4. Are there plans to expand eteplirsen’s approved indications?
Sarepta is pursuing approvals for additional exon-skipping drugs targeting different mutations, which may indirectly benefit eteplirsen’s market by improving overall disease management.

5. How can Sarepta optimize eteplirsen’s financial potential?
By strengthening clinical data, engaging with payers to improve reimbursement, expanding indications, and collaborating with patient groups, Sarepta can maximize eteplirsen’s market impact.

---

References

1. [1] Sarepta Therapeutics. Eteplirsen (EXONDYS 51) Prescribing Information. 2016.
2. [2] U.S. Food and Drug Administration. FDA Approves First Drug for Duchenne Muscular Dystrophy. 2016.
3. [3] Osborn, A. “Eteplirsen Clinical Efficacy Under Scrutiny Amid Market Challenges,” Pharma Business Review. 2022.
4. [4] GlobalData. Duchenne Muscular Dystrophy Market Forecast. 2023.