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 nine patents protecting this compound. Additional information is available in the individual branded drug profile pages.

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

One supplier is listed for this compound.

Summary for ETEPLIRSEN

International Patents:	127
US Patents:	9
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	206488-001	Sep 19, 2016		RX	Yes	Yes			Y	Y
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	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			Y	Y

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	206488-001	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

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/004355 Treatment of Duchenne muscular dystrophy.	Refused	no	no	yes		2018-12-06

International Patents for ETEPLIRSEN

Country	Patent Number	Title	Estimated Expiration
Japan	6885620
European Patent Office	3400948	PROCÉDÉS ET MOYENS POUR SAUTER EFFICACEMENT AU MOINS L`UN DES EXONS SUIVANTS DU GÈNE DE LA DYSTROPHIE MUSCULAIRE HUMAINE DE DUCHENNE : 43, 46, 50 À 53 (METHODS AND MEANS FOR EFFICIENT SKIPPING OF AT LEAST ONE OF THE FOLLOWING EXONS OF THE HUMAN DUCHENNE MUSCULAR DYSTROPHY GENE: 43, 46, 50- 53)
Israel	278912	תכשירים משופרים לטיפול דיסטרופיה של השרירים (Improved compositions for treating muscular dystrophy)
Canada	2741629	PROCEDES ET MOYENS D'INDUCTION DU SAUT DE L'EXON 45 DANS L'ARN PRE-MESSAGER DU GENE DE LA DYSTROPHIE MUSCULAIRE DE DUCHENNE (METHODS AND MEANS FOR EFFICIENT SKIPPING OF EXON 45 IN DUCHENNE MUSCULAR DYSTROPHY PRE-MRNA)

ETEPLIRSEN Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Eteplirsen

Introduction to Eteplirsen

Eteplirsen, developed by Sarepta Therapeutics, is a groundbreaking drug designed to treat Duchenne Muscular Dystrophy (DMD), a severe genetic disorder affecting muscle function. The drug works by enabling the skipping of exon 51, a genetic mutation that affects over 13% of DMD patients, allowing for the production of a truncated but partially functional dystrophin protein[2].

Approval and Controversy

Eteplirsen was approved by the FDA in September 2016, despite controversy surrounding its clinical trials. The approval was granted after a lengthy review process, including a Priority Review status, and despite reservations from the FDA's advisory board. The decision was criticized for being based on limited data from a small patient group, sparking debates about its efficacy and safety[2][4].

Clinical Benefits and Impact

Despite the controversy, eteplirsen has shown clinical benefits, particularly in delaying disease progression. Studies have indicated that eteplirsen treatment is associated with improvements in lung function, delays in time to loss of ambulation, and various quality of life measures compared to natural history controls or standard care[1].

Market Size and Growth

The Duchenne Muscular Dystrophy (DMD) drugs market, driven significantly by exon-skipping therapies like eteplirsen, is experiencing robust growth. As of 2023, the market size was valued at USD 2.3 billion and is projected to reach USD 5.5 billion by 2032, with a Compound Annual Growth Rate (CAGR) of 9.96% during the forecast period[3].

Revenue and Financial Performance

Sarepta Therapeutics has seen significant financial growth driven by the success of eteplirsen, now marketed as Exondys 51. In the first quarter of 2024, Sarepta reported net product revenue of $359.5 million, a substantial increase from the previous year. This growth is attributed to the increasing demand for Exondys 51, as well as collaboration revenues and contract manufacturing revenues associated with commercial supply to partners like Roche[1].

Pricing and Affordability

Eteplirsen is priced at approximately $300,000 per patient per year, varying based on patient weight. While this price is significant, it reflects the drug's status as a rare disease treatment. The high cost has raised questions about its affordability and whether the clinical benefits justify the expense[2].

Geographical Market Presence

The DMD drugs market, including eteplirsen, has a strong presence in North America, which accounts for the largest market share. This is due to the high investment in research and development, emphasis on personalized medicines, and collaborations between pharmaceutical companies in this region[3].

Competitive Landscape

Eteplirsen is part of a growing landscape of DMD treatments, including other exon-skipping therapies like golodirsen, which targets exon 53 mutations. Together, these therapies cover approximately 30% of DMD patients with amenable mutations. The market is also seeing advancements in combination therapies and personalized medicine approaches[3].

Future Outlook and Innovations

The future of eteplirsen and the broader DMD market is promising, with ongoing innovations in treatment surgeries and the development of new therapies. There is a growing emphasis on combination therapies and personalized medicine, which is expected to drive further growth in the market. Sarepta Therapeutics continues to invest in research and development, aiming to expand its portfolio beyond eteplirsen[3].

Challenges and Criticisms

Despite its clinical benefits, eteplirsen faces challenges related to its approval process and the need for stronger evidence of efficacy. Critics argue that the drug's approval set a precedent for lower standards of evidence, and there are ongoing debates about its safety and long-term effectiveness[2][4].

Patient and Market Impact

The approval and availability of eteplirsen have significantly impacted DMD patients and their families, offering a treatment option where few existed before. However, the high cost and disputed efficacy have raised concerns about accessibility and value for money. Annual net sales for eteplirsen and other targeted DMD therapies have increased dramatically, from $7 million in 2016 to $879 million in 2022, reflecting the drug's market impact[5].

Key Takeaways

• Approval and Controversy: Eteplirsen was approved in 2016 despite controversy over its clinical trial data.
• Clinical Benefits: The drug has shown improvements in lung function, delays in loss of ambulation, and quality of life measures.
• Market Growth: The DMD drugs market is projected to grow to USD 5.5 billion by 2032.
• Financial Performance: Sarepta Therapeutics has seen significant revenue growth driven by eteplirsen.
• Pricing and Affordability: The drug is priced at approximately $300,000 per patient per year, raising affordability concerns.
• Future Outlook: The market is driven by innovations in treatment and personalized medicine approaches.

Frequently Asked Questions

Q: What is eteplirsen and how does it work?

A: Eteplirsen is a drug designed to treat Duchenne Muscular Dystrophy (DMD) by enabling the skipping of exon 51, allowing for the production of a truncated but partially functional dystrophin protein.

Q: Why was the approval of eteplirsen controversial?

A: The approval was controversial due to the limited data from a small patient group and reservations from the FDA's advisory board.

Q: How much does eteplirsen cost?

A: Eteplirsen is priced at approximately $300,000 per patient per year, varying based on patient weight.

Q: What is the projected growth of the DMD drugs market?

A: The DMD drugs market is expected to reach USD 5.5 billion by 2032, with a CAGR of 9.96% during the forecast period.

Q: What are the key benefits of eteplirsen for DMD patients?

A: Eteplirsen has been associated with improvements in lung function, delays in time to loss of ambulation, and various quality of life measures.

Sources:

1. Sarepta Therapeutics Announces First Quarter 2024 Financial Results.
2. eteplirsen: a controversial approval - eCampusOntario Pressbooks.
3. Duchenne Muscular Dystrophy Drugs Market Size to Reach USD 5.5 Billion by 2032 - BioSpace.
4. FDA Approval Document for Eteplirsen.
5. Spending on Targeted Therapies for Duchenne Muscular Dystrophy - JAMA Network.