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VYONDYS 53 Drug Patent Profile

When do Vyondys 53 patents expire, and when can generic versions of Vyondys 53 launch?

Vyondys 53 is a drug marketed by Sarepta Theraps Inc and is included in one NDA. There are eight patents protecting this drug.

This drug has thirty patent family members in thirteen countries.

The generic ingredient in VYONDYS 53 is golodirsen. One supplier is listed for this compound. Additional details are available on the golodirsen profile page.

DrugPatentWatch^® Generic Entry Outlook for Vyondys 53

Vyondys 53 was eligible for patent challenges on December 12, 2023.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be December 12, 2026. This may change due to patent challenges or generic licensing.

There have been two patent litigation cases involving the patents protecting this drug, indicating strong interest in generic launch. Recent data indicate that 63% of patent challenges are decided in favor of the generic patent challenger and that 54% of successful patent challengers promptly launch generic drugs.

Indicators of Generic Entry

Summary for VYONDYS 53

International Patents:	30
US Patents:	8
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for VYONDYS 53
What excipients (inactive ingredients) are in VYONDYS 53?	VYONDYS 53 excipients list
DailyMed Link:	VYONDYS 53 at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for VYONDYS 53

Constraining patent/regulatory exclusivity:

INDICATED FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS WHO HAVE A CONFIRMED MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 53 SKIPPING

NDA:

211970

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.

Pharmacology for VYONDYS 53

Drug Class	Antisense Oligonucleotide
Physiological Effect	Increased Protein Synthesis

US Patents and Regulatory Information for VYONDYS 53

VYONDYS 53 is protected by eight US patents and two FDA Regulatory Exclusivities.

This potential generic entry date is based on INDICATED FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS WHO HAVE A CONFIRMED MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 53 SKIPPING.

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	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019		RX	Yes	Yes	10,266,827
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019		RX	Yes	Yes	10,421,966		Y	Y
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019		RX	Yes	Yes	10,227,590		Y	Y
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019		RX	Yes	Yes	9,994,851		Y	Y
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019		RX	Yes	Yes	10,995,337		Y
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019		RX	Yes	Yes	RE47691		Y

Expired US Patents for VYONDYS 53

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019	10,533,174
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019	9,416,361

International Patents for VYONDYS 53

See the table below for patents covering VYONDYS 53 around the world.

Country	Patent Number	Title	Estimated Expiration
European Patent Office	2351839
Australia	2004285494
Cyprus	1120409
World Intellectual Property Organization (WIPO)	2006033933
Australia	2007223776
South Korea	20020097241

VYONDYS 53 Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for VYONDYS 53

Introduction to VYONDYS 53

VYONDYS 53, developed by Sarepta Therapeutics, is a groundbreaking treatment for Duchenne muscular dystrophy (DMD) patients with a specific mutation in the dystrophin gene that can be treated by skipping exon 53. This drug has been approved under accelerated approval based on its ability to increase dystrophin production in skeletal muscle[2].

Market Context: Duchenne Muscular Dystrophy Drugs

The Duchenne muscular dystrophy drugs market is experiencing significant growth, driven by innovations in treatment and surgeries. 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 period 2024-2032. This growth is largely attributed to advancements in exon-skipping therapies, such as VYONDYS 53, and the increasing emphasis on personalized medicine and combination therapies[4].

Sarepta Therapeutics' Financial Performance

Sarepta Therapeutics has seen substantial financial growth, particularly driven by the success of its PMO (phosphorodiamidate morpholino oligomer) products, including VYONDYS 53. In the fourth quarter of 2023, Sarepta reported a 55% increase in net product revenue compared to the same quarter in 2022, with full-year revenue increasing by 36%. This robust performance is a testament to the increasing demand for these therapies[1].

Revenue Contributions from VYONDYS 53

VYONDYS 53, along with other PMO products like EXONDYS 51 and AMONDYS 45, has been a key contributor to Sarepta's revenue growth. The successful launch and continued adoption of these therapies have significantly boosted the company's net product revenues. For instance, in the third quarter of 2024, Sarepta's net product revenues totaled $429.8 million, a 39% increase over the same quarter in the prior year[3].

Geographical Market Presence

The market for Duchenne muscular dystrophy drugs, including VYONDYS 53, is predominantly driven by regions such as North America, Asia Pacific, Europe, Latin America, and the Middle East and Africa. North America accounts for the largest market share, largely due to the high investment in research and development and the emphasis on personalized medicines[4].

Clinical and Regulatory Landscape

VYONDYS 53 has been approved under accelerated approval, contingent upon verification of clinical benefit in confirmatory trials. The drug's efficacy is based on its ability to increase dystrophin production, which aims to slow disease progression. However, it is crucial to monitor kidney function in patients, as kidney toxicity has been observed with similar drugs, although not in clinical studies with VYONDYS 53 itself[2][3].

Side Effects and Monitoring

Patients treated with VYONDYS 53 may experience various side effects, including headache, fever, fall, abdominal pain, and infections. Regular monitoring of kidney function is essential, involving urine and blood tests to mitigate the risk of kidney toxicity. Healthcare providers play a critical role in managing these side effects and ensuring ongoing patient safety[2].

Patient Support and Access

Sarepta Therapeutics offers a patient support program called SareptAssist to help patients navigate the process of starting and staying on VYONDYS 53. This program provides valuable information and assistance, ensuring that patients have access to the treatment they need[2].

Competitive Landscape

The market for DMD treatments is becoming increasingly competitive, with several pharmaceutical companies investing in research and development. Sarepta Therapeutics, however, has established a strong presence with its PMO products and the recent success of its gene therapy, ELEVIDYS. The company's decision to discontinue the SRP-5051 (vesleteplirsen) development program reflects its strategic focus on the most promising therapeutic avenues[3].

Future Outlook

Given the projected growth of the DMD drugs market and the increasing demand for exon-skipping therapies, VYONDYS 53 is poised for continued success. Sarepta Therapeutics' strong financial performance and its commitment to innovation and patient support position the company well to capitalize on this growing market.

Key Takeaways

• Market Growth: The DMD drugs market is expected to reach USD 5.5 billion by 2032, driven by innovations in exon-skipping therapies.
• Financial Performance: Sarepta Therapeutics has seen significant revenue growth, with a 55% increase in net product revenue in the fourth quarter of 2023.
• Clinical Efficacy: VYONDYS 53 increases dystrophin production, aiming to slow DMD progression, but requires careful monitoring of kidney function.
• Patient Support: SareptAssist provides essential support for patients starting and maintaining treatment with VYONDYS 53.
• Competitive Landscape: Sarepta Therapeutics maintains a strong market position with its PMO products and gene therapies.

Frequently Asked Questions

Q: What is VYONDYS 53 used for? A: VYONDYS 53 is used to treat patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 53[2].

Q: What are the common side effects of VYONDYS 53? A: Common side effects include headache, fever, fall, abdominal pain, and infections. Regular monitoring of kidney function is also necessary[2].

Q: How does Sarepta Therapeutics support patients on VYONDYS 53? A: Sarepta offers the SareptAssist program to help patients navigate the treatment process and ensure access to the medication[2].

Q: What is the projected market size for DMD drugs by 2032? A: The market is expected to reach USD 5.5 billion by 2032, with a CAGR of 9.96% during 2024-2032[4].

Q: Why is kidney function monitoring important for patients on VYONDYS 53? A: Although kidney toxicity was not observed in clinical studies with VYONDYS 53, it has been seen with similar drugs. Regular monitoring is crucial to mitigate this risk[2][3].

Cited Sources

1. Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2023 Financial Results and Recent Corporate Developments.
2. VYONDYS 53 Insurance Coverage & Assistance with SareptAssist.
3. Sarepta Therapeutics Announces Third Quarter 2024 Financial Results and Recent Corporate Developments.
4. Duchenne Muscular Dystrophy Drugs Market Size to Reach USD 5.5 Billion by 2032.