VILTEPSO Drug Patent Profile

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

Viltepso is a drug marketed by Nippon Shinyaku and is included in one NDA. There are two patents protecting this drug.

This drug has fifty-six patent family members in twenty countries.

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

DrugPatentWatch^® Generic Entry Outlook for Viltepso

Viltepso was eligible for patent challenges on August 12, 2024.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be August 31, 2031. This may change due to patent challenges or generic licensing.

Indicators of Generic Entry

Summary for VILTEPSO

International Patents:	56
US Patents:	2
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	1
Drug Prices:	Drug price information for VILTEPSO
What excipients (inactive ingredients) are in VILTEPSO?	VILTEPSO excipients list
DailyMed Link:	VILTEPSO at DailyMed

See drug prices for VILTEPSO

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

NDA:

212154

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.

US Patents and Regulatory Information for VILTEPSO

VILTEPSO is protected by two US patents and two FDA Regulatory Exclusivities.

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
Nippon Shinyaku	VILTEPSO	viltolarsen	SOLUTION;INTRAVENOUS	212154-001	Aug 12, 2020		RX	Yes	Yes			Y	Y
Nippon Shinyaku	VILTEPSO	viltolarsen	SOLUTION;INTRAVENOUS	212154-001	Aug 12, 2020		RX	Yes	Yes
Nippon Shinyaku	VILTEPSO	viltolarsen	SOLUTION;INTRAVENOUS	212154-001	Aug 12, 2020		RX	Yes	Yes			Y	Y
Nippon Shinyaku	VILTEPSO	viltolarsen	SOLUTION;INTRAVENOUS	212154-001	Aug 12, 2020		RX	Yes	Yes

International Patents for VILTEPSO

When does loss-of-exclusivity occur for VILTEPSO?

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

Hungary

Japan

Lithuania

Poland

Portugal

Russian Federation

San Marino

Serbia

Slovenia

South Korea

Spain

Taiwan

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 VILTEPSO around the world.

Country	Patent Number	Title	Estimated Expiration
China	103154245	Antisense nucleic acid
European Patent Office	3018211	ACIDES NUCLÉIQUES ANTISENS (ANTISENSE NUCLEIC ACIDS)
Japan	2014054250	ANTISENSE NUCLEIC ACIDS
Japan	2023036865
Japan	6647430
Poland	3018211
Serbia	59361

Market Dynamics and Financial Trajectory for Viltepso

Introduction to Viltepso

Viltepso, developed and marketed by NS Pharma, is a groundbreaking treatment for Duchenne muscular dystrophy (DMD), a severe genetic disorder affecting muscle function. Approved in Japan in March 2020 and in the U.S. in August 2020, Viltepso has been at the forefront of nucleic acid medicine research.

Market Context

Duchenne Muscular Dystrophy Market

The DMD market is rapidly evolving, driven by innovative therapies. As of 2023, the DMD market was valued at USD 2,150 million and is expected to grow significantly through 2034 with new therapies[3].

Competitive Landscape

Viltepso competes in a market dominated by Sarepta Therapeutics, which has four approved antisense oligonucleotide products: EXONDYS 51, VYONDYS 53, AMONDYS 45, and ELEVIDYS. These products collectively treat around 30% of all DMD cases. Despite this competition, Viltepso holds a unique position, particularly in the Japanese market where it is the only approved drug for DMD patients[3].

Regulatory Approvals and Expansion

Initial Approvals

Viltepso received accelerated approval from the U.S. FDA in August 2020, just a week after the application was submitted. This approval was based on Phase 2 study results showing an increase in dystrophin levels, which was likely to predict clinical benefit[1][5].

Global Expansion Efforts

Following its approval in the U.S. and Japan, NS Pharma has been working to secure regulatory approvals in other regions. The company has filed a submission for regulatory approval in China and is in discussions with European regulators. These efforts aim to make Viltepso accessible to DMD patients worldwide[1].

Clinical Trials and Efficacy

Phase 2 and Phase 3 Trials

The Phase 2 study of Viltepso showed promising results, with an increase in dystrophin levels and a delay in motor function decline in boys with DMD. However, the confirmatory Phase 3 RACER53 trial did not meet its primary endpoint, showing no significant improvement in motor function over placebo. Despite this, the treatment was well-tolerated, and further analyses are being conducted to identify factors that may have influenced the results[5].

Safety and Tolerability

Viltepso has demonstrated a favorable safety profile, with mild to moderate adverse events reported in both Phase 2 and Phase 3 studies. There were no side effects leading to treatment discontinuation[5].

Market Access and Sales

U.S. Market

In the U.S., Viltepso is marketed by NS Pharma's subsidiary, NS Pharma, Inc. (NSP), based in New Jersey. The market access team plays a crucial role in negotiating with insurers and resolving other related issues to ensure patient access to the drug. Despite the challenges posed by the COVID-19 pandemic, the team's efforts have been instrumental in maintaining operations and building trust with patients and healthcare providers[1].

Revenue and Financial Performance

Viltepso has contributed significantly to NS Pharma's revenue. For the first half of FY2022, the company reported increased revenues from pharmaceuticals, including Viltepso. The forecast for FY2022 indicated that sales of Viltepso, along with other products, were expected to exceed previous projections, leading to revised annual forecasts for revenue, operating profit, and profit before tax[2].

Financial Projections and Outlook

Revenue Growth

NS Pharma's financial reports show a positive trend in revenue growth, partly driven by the sales of Viltepso. For FY2022, the company projected an increase in revenue from pharmaceuticals, including Viltepso, which contributed to the overall revenue growth of 2.6% compared to the previous year[2].

Operating Profit and Profitability

Despite the challenges in the Phase 3 trial, NS Pharma's operating profit and profitability are expected to remain stable. The company has revised its annual forecasts, indicating a slight decrease in operating profit but an overall positive outlook for the financial year[2].

Global Development and Strategic Partnerships

Ongoing Clinical Trials

NS Pharma is coordinating global Phase III clinical studies and other clinical trials for Viltepso. These ongoing trials are crucial for gathering more clinical data and potentially expanding the drug's approval in other markets[1].

Strategic Partnerships

The company is considering optimal approaches in each market based on its in-house resources and strategic partnerships. This strategic approach is aimed at accelerating regulatory approvals and market access in various regions[1].

Key Takeaways

• Regulatory Approvals: Viltepso has received approvals in Japan and the U.S. and is seeking approvals in China and Europe.
• Clinical Trials: While the Phase 3 RACER53 trial did not meet its primary endpoint, earlier Phase 2 studies showed promising results.
• Market Access: NS Pharma's market access team plays a critical role in ensuring patient access to Viltepso in the U.S.
• Financial Performance: Viltepso has contributed to NS Pharma's revenue growth, with positive financial projections despite the challenges in the Phase 3 trial.
• Global Expansion: Ongoing efforts to secure approvals and partnerships are aimed at expanding Viltepso's reach globally.

FAQs

What is Viltepso used for?

Viltepso is used to treat Duchenne muscular dystrophy (DMD), a genetic disorder that affects muscle function. It is specifically designed for patients with a mutation amenable to exon 53 skipping.

Who developed and markets Viltepso?

Viltepso was developed and is marketed by NS Pharma.

What were the results of the Phase 3 RACER53 trial for Viltepso?

The Phase 3 RACER53 trial did not meet its primary endpoint, showing no significant improvement in motor function over placebo. However, the treatment was well-tolerated, and further analyses are being conducted.

How is Viltepso administered?

Viltepso is administered as a once-weekly infusion directly into the bloodstream.

What is the current market status of Viltepso?

Viltepso is approved in Japan and the U.S. and is in the process of seeking regulatory approvals in China and Europe.

What are the financial projections for Viltepso?

Despite the challenges in the Phase 3 trial, NS Pharma's financial projections indicate a positive outlook, with expected revenue growth and stable profitability.

Sources

1. Nippon Shinyaku Co., Ltd. - Getting Viltepso to the Patients Who Need It [PDF]
2. Nippon Shinyaku Co., Ltd. - Outline of Consolidated Financial Results for the 2nd Quarter Ended ... [PDF]
3. DelveInsight - Duchenne Muscular Dystrophy: Innovations Beyond Exon-Skipping
4. Nippon Shinyaku Co., Ltd. - Leading Product Revenue | Business/Financial Information
5. Muscular Dystrophy News - Viltepso fails main goal in confirmatory Phase 3 study