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EXONDYS 51 Drug Patent Profile

Which patents cover Exondys 51, and when can generic versions of Exondys 51 launch?

Exondys 51 is a drug marketed by Sarepta Theraps Inc and is included in one NDA. There are nine patents protecting this drug.

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

The generic ingredient in EXONDYS 51 is eteplirsen. One supplier is listed for this compound. Additional details are available on the eteplirsen profile page.

DrugPatentWatch^® Generic Entry Outlook for Exondys 51

Exondys 51 was eligible for patent challenges on September 19, 2020.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be October 27, 2028. This may change due to patent challenges or generic licensing.

Indicators of Generic Entry

Summary for EXONDYS 51

International Patents:	127
US Patents:	9
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Clinical Trials:	2
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for EXONDYS 51
What excipients (inactive ingredients) are in EXONDYS 51?	EXONDYS 51 excipients list
DailyMed Link:	EXONDYS 51 at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for EXONDYS 51

Constraining patent/regulatory exclusivity:

9,243,245

NDA:

206488

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 EXONDYS 51

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

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

See all EXONDYS 51 clinical trials

Pharmacology for EXONDYS 51

Drug Class

Antisense Oligonucleotide

US Patents and Regulatory Information for EXONDYS 51

EXONDYS 51 is protected by fifteen US patents.

This potential generic entry date is based on patent 9,243,245.

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

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	RE47769		Y
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016		RX	Yes	Yes	10,781,451		Y	Y
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	Sep 19, 2016		RX	Yes	Yes	9,506,058
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016		RX	Yes	Yes	RE47751

Expired US Patents for EXONDYS 51

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	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	206488-001	Sep 19, 2016	9,416,361

EU/EMA Drug Approvals for EXONDYS 51

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 EXONDYS 51

When does loss-of-exclusivity occur for EXONDYS 51?

Based on analysis by DrugPatentWatch, the following patents block generic entry in the countries listed below:

Australia

Canada

China

Croatia

Cyprus

Denmark

European Patent Office

Hong Kong

Hungary

Israel

Japan

New Zealand

Poland

Portugal

Slovenia

Spain

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

See the table below for additional patents covering EXONDYS 51 around the world.

Country	Patent Number	Title	Estimated Expiration
China	113633787	改进的用于治疗肌营养不良的组合物 (Improved compositions for treating muscular dystrophy)
Canada	2584599
New Zealand	732507	Improved compositions for treating muscular dystrophy
Japan	5786109

EXONDYS 51 Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for EXONDYS 51

Introduction

EXONDYS 51, developed by Sarepta Therapeutics, is a groundbreaking treatment for Duchenne Muscular Dystrophy (DMD), a rare and debilitating genetic disorder. Since its approval in 2016, the drug has navigated a complex landscape of market dynamics and financial performance. Here, we delve into the key aspects of its market and financial trajectory.

Approval and Initial Market Reception

EXONDYS 51 was approved by the FDA in September 2016 for DMD patients with a confirmed mutation amenable to exon 51 skipping. Despite the controversy surrounding its accelerated approval due to limited clinical evidence, the drug quickly gained traction in the market. This approval marked a significant milestone, as it was the first FDA-approved treatment for DMD[4].

Early Financial Performance

In its first full year on the market, EXONDYS 51 generated $155 million in net product sales for Sarepta Therapeutics. This figure was indicative of the strong demand for new treatment options in the DMD market. The initial success was a promising start, setting the stage for future growth[1].

Market Growth and Expansion

The demand for EXONDYS 51 continued to grow, contributing significantly to Sarepta's revenue. By the first quarter of 2019, net revenues from EXONDYS 51 had increased, reflecting the ongoing rise in demand. For the three months ended March 31, 2019, Sarepta recorded net revenues of $87.0 million, up from $64.6 million in the same period of 2018. This increase was primarily driven by the growing adoption of EXONDYS 51 in the U.S.[2].

Competitive Landscape

The DMD market, though small, is highly competitive and dominated by a few key players. EXONDYS 51, along with other Sarepta therapies like VYONDYS 53, has carved out a significant niche. The approval of VYONDYS 53 further solidified Sarepta's position in the market, demonstrating the company's commitment to addressing the high unmet needs in DMD treatment[4].

Financial Performance Over the Years

Quarterly and Annual Revenues

By the second quarter of 2023, Sarepta's net product revenues from EXONDYS 51, along with other treatments like AMONDYS 45 and VYONDYS 53, totaled $239.0 million. This represented a 13% increase over the same quarter in the prior year. The full-year 2020 saw net product revenues of $455.9 million, an increase of $75.1 million from 2019, primarily due to the continued demand for EXONDYS 51 and the launch of VYONDYS 53[3][5].

Cost and Operating Expenses

The success of EXONDYS 51 has also led to increased costs for Sarepta. For instance, the cost of sales (excluding amortization of in-licensed rights) rose to $12.1 million in the first quarter of 2019, up from $5.6 million in the same period of 2018. This increase was due to higher product costs, royalty payments to BioMarin Pharmaceuticals, and an inventory write-off related to certain batches of product not meeting quality specifications[2].

Pipeline Development and Future Growth

Sarepta is not resting on its laurels; the company is aggressively expanding its pipeline with RNA-targeting and gene therapy technologies. The micro-dystrophin gene therapy, for example, has shown strong promise and is expected to position Sarepta as a leader in the DMD treatment field. Investment bank Leerink estimates that worldwide sales of Sarepta's DMD treatments could reach around $900 million by 2027[1].

Regulatory and Market Milestones

The FDA's accelerated approval of EXONDYS 51 and subsequent approvals of other Sarepta therapies have set important precedents. The approval of ELEVIDYS (delandistrogene moxeparvovec-rokl) in June 2023, for which Sarepta received a Rare Pediatric Disease Priority Review Voucher (PRV), further underscores the company's regulatory success and market impact[3].

Impact on Sarepta's Valuation

The success of EXONDYS 51 has significantly boosted Sarepta's valuation. The drug's performance, along with positive data from other pipeline treatments, has added billions to the company's market value. For instance, positive data from just three patients treated with a new gene therapy added $2.5 billion to Sarepta's value, highlighting the market's confidence in the company's scientific advancements[1].

Key Takeaways

• Strong Initial Reception: EXONDYS 51 generated $155 million in its first full year, indicating strong market demand.
• Continuous Growth: Net revenues from EXONDYS 51 have consistently increased, contributing to Sarepta's overall revenue growth.
• Competitive Position: Sarepta has established itself as a leader in the DMD treatment market with EXONDYS 51 and other therapies.
• Pipeline Expansion: The company is investing heavily in RNA-targeting and gene therapy technologies to further its market dominance.
• Regulatory Success: FDA approvals and the receipt of a PRV have bolstered Sarepta's regulatory and market standing.

FAQs

Q: What is EXONDYS 51 used for?

A: EXONDYS 51 is used to treat Duchenne Muscular Dystrophy (DMD) in patients with a confirmed mutation amenable to exon 51 skipping.

Q: When was EXONDYS 51 approved by the FDA?

A: EXONDYS 51 was approved by the FDA in September 2016.

Q: How much revenue did EXONDYS 51 generate in its first full year on the market?

A: EXONDYS 51 generated $155 million in net product sales during its first full year on the market.

Q: What are the projected sales for Sarepta's DMD treatments by 2027?

A: Investment bank Leerink estimates that worldwide sales of Sarepta's DMD treatments could reach around $900 million by 2027.

Q: How has the success of EXONDYS 51 impacted Sarepta's valuation?

A: The success of EXONDYS 51, along with positive data from other pipeline treatments, has significantly boosted Sarepta's valuation, adding billions to the company's market value.

Sources

1. BioPharma Dive: "3 patients add $2.5B to Sarepta's value"
2. Sarepta Therapeutics: "Sarepta Therapeutics Announces First Quarter 2019 Financial Results"
3. Sarepta Therapeutics: "Sarepta Therapeutics Announces Second Quarter 2023 Financial Results and Recent Corporate Developments"
4. DelveInsight: "Sarepta Therapeutics Advances DMD Pipeline"
5. GlobeNewswire: "Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2020 Financial Results and Recent Corporate Developments"