Golodirsen - Generic Drug Details

What are the generic drug sources for golodirsen and what is the scope of freedom to operate?

Golodirsen 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.

Golodirsen has thirty patent family members in thirteen countries.

One supplier is listed for this compound.

Summary for golodirsen

International Patents:	30
US Patents:	8
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Clinical Trials:	4
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for golodirsen
What excipients (inactive ingredients) are in golodirsen?	golodirsen excipients list
DailyMed Link:	golodirsen at DailyMed

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

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

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 golodirsen

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

Sponsor	Phase
Rare Disease Research, LLC	Phase 4
Sarepta Therapeutics, Inc.	Phase 4
Sarepta Therapeutics, Inc.	Phase 2

See all golodirsen clinical trials

Pharmacology for golodirsen

Drug Class	Antisense Oligonucleotide
Physiological Effect	Increased Protein Synthesis

Anatomical Therapeutic Chemical (ATC) Classes for golodirsen

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 golodirsen

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			Y	Y
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019		RX	Yes	Yes
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019		RX	Yes	Yes			Y	Y
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019		RX	Yes	Yes			Y
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019		RX	Yes	Yes			Y	Y
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019		RX	Yes	Yes			Y	Y
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019		RX	Yes	Yes

Expired US Patents for golodirsen

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
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019

International Patents for golodirsen

Country	Patent Number	Title	Estimated Expiration
European Patent Office	4272748	OLIGONUCLÉOTIDES ANTISENS POUR INDUIRE L'OMISSION DE L'EXON ET LEURS PROCÉDÉS D'UTILISATION (ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF)
Spain	2361325
Austria	E498685
European Patent Office	4047096	OLIGONUCLÉOTIDES ANTISENS POUR INDUIRE L'OMISSION DE L'EXON ET LEURS PROCÉDÉS D'UTILISATION (ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF)
European Patent Office	2500430	Oligonucléotides antisens permettant d'induire un saut d'exon et leurs procédés d'utilisation (Antisense oligonucleotides for inducing exon skipping and methods of use thereof)
Hong Kong	1245325	用於誘導外顯子跳躍的反義寡核苷酸及其使用方法 (ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF)
Portugal	1766010

Market Dynamics and Financial Trajectory for Golodirsen

Last updated: July 28, 2025

Introduction

Golodirsen, marketed under the brand name Vyondys 53, is a groundbreaking antisense oligonucleotide developed by Sarepta Therapeutics. Approved by the U.S. Food and Drug Administration (FDA) in December 2019, golodirsen offers a targeted therapeutic option for rare genetic disorders, specifically Duchenne muscular dystrophy (DMD) caused by specific genetic mutations. Its market prospects are shaped by an intricate interplay of clinical efficacy, regulatory landscape, competitive dynamics, and broader healthcare trends. This analysis offers a comprehensive view of the evolving market landscape and financial trajectory of golodirsen within the biopharmaceutical industry.

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Pharmaceutical Profile and Clinical Context

Golodirsen functions as a phosphorodiamidate morpholino oligomer (PMO) that induces exon 53 skipping during pre-mRNA splicing, restoring the production of dystrophin protein in certain DMD patients. DMD is a severe X-linked genetic disorder affecting approximately 1 in 3,500 male births globally, characterized by progressive muscle degeneration and loss of ambulation [1].

The drug’s approval was based on surrogate endpoints demonstrating increased dystrophin production. While clinical improvements remain modest, golodirsen represents an innovative therapeutic class targeting genetic mutations at the mRNA level. Its niche application underscores both its clinical relevance and its potential limitations in broader market adoption.

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Market Dynamics

1. Epidemiological and Patient Population Factors

The primary target population for golodirsen comprises DMD patients with confirmed mutations amenable to exon 53 skipping, accounting for approximately 8-10% of the DMD population [2]. Given the rarity of the condition, the total addressable market (TAM) remains inherently limited, with estimates suggesting around 1,000 to 1,500 eligible new cases annually in the U.S. alone.

Globally, this population expands modestly, influenced by genetic screening and diagnostic advances. As awareness improves, the identification of eligible patients may increase, although the overall prevalence constrains market ceiling.

2. Competitive Landscape

Golodirsen operates within a highly competitive space characterized by the development and commercialization of multiple exon-skipping therapies. Sarepta’s primary competitor, BioMarin Pharmaceutical’s casimersen, targets exon 45 skipping, covering a different subset of DMD patients but overlapping in the broader exon-skipping approach [3].

Other potential competitors include emerging therapies employing gene editing, cell therapy, or alternative antisense modalities. The lack of direct, large-scale head-to-head trials emphasizes a fragmented competitive environment, where approval, pricing, and reimbursement policies greatly influence market share.

3. Regulatory and Reimbursement Environment

While the initial FDA approval prioritized accelerated pathways based on surrogate endpoints, ongoing confirmatory studies are critical for market expansion and payer confidence. The conditional nature of initial approvals entails risks related to post-market evidence generation.

Reimbursement strategies significantly impact patient access. Historically, orphan drugs like golodirsen face price challenges, with costs exceeding $300,000 annually. Payers are scrutinizing cost-effectiveness, especially as multiple exon-skipping options emerge. Reimbursement negotiations are thus central to commercial success.

4. Manufacturing and Supply Chain Dynamics

Supply constraints can influence market penetration, especially given the complexity of oligonucleotide manufacturing. Sarepta’s ability to scale production efficiently and maintain quality standards remains pivotal for sustained revenue growth.

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Financial Trajectory

1. Revenue Generation and Market Penetration

Sarepta initially launched golodirsen in the U.S. with a focus on specialized neuromuscular centers. Estimated first-year sales hovered around $50 million, reflecting cautious adoption owing to the drug’s niche application and reimbursement hurdles [4]. As awareness grows and clinical data accumulate, sales are projected to increase, with analysts estimating a compounded annual growth rate (CAGR) of approximately 15-20% over the next five years.

However, the total revenue potential remains capped by the small patient population and payer constraints. The drug’s high per-patient pricing sustains revenues but attracts scrutiny amid broader healthcare cost containment efforts.

2. R&D Investment and Pipeline Impact

Investments in refining exon-skipping technology and expanding indications influence Sarepta’s long-term financial outlook. The company’s pipeline includes next-generation therapies and broader indications, which could augment golodirsen-related revenues if they reach commercialization.

Sarepta’s financial reports reveal ongoing R&D expenditure focused on evidentiary studies, manufacturing optimization, and companion diagnostics, vital for expanding approval subsets [5].

3. Cost Structure and Profitability

Given its orphan drug designation, golodirsen benefits from expedited development incentives and pricing premiums. However, manufacturing complexities add variable costs.

Profit margins are currently modest owing to high R&D and commercialization expenses. Long-term profitability hinges on stable sales growth, continued reimbursement success, and potential licensing or partnership agreements.

4. Market Penetration Strategies

Sarepta employs targeted education campaigns for clinicians and patient advocacy groups. Access programs and expanded label claims could broaden patient eligibility, thus enhancing revenue. Collaborations with payers to demonstrate cost-effectiveness are also integral to sustaining market share.

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Future Outlook and Key Drivers

1. Regulatory Milestones

The success of ongoing confirmatory trials will determine future approval extensions, potentially covering broader mutations or multiple exon skipping. Positive data could catalyze label expansion, boosting sales.

2. Evolving Therapeutic Landscape

Emerging gene therapies, such as Crispr-based editing, threaten to disrupt the exon-skipping paradigm. Nonetheless, oligonucleotide therapies like golodirsen retain a competitive edge due to their established safety profiles and manufacturing familiarity.

3. Pricing, Reimbursement, and Market Acceptance

Pricing strategies balancing innovation incentives with payer affordability will shape financial trajectories. Early engagement with payers and demonstration of clinical benefit are critical for widespread reimbursement.

4. Broader Disease Indications

Research into extending antisense oligonucleotide applicability into other neuromuscular or genetic disorders could unlock new revenue streams, exponentially improving the market outlook.

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Key Takeaways

• Limited but Critical Market: Golodirsen’s niche targeting exon 53 mutations restricts maximum revenue but offers substantial value for enrolled patients.
• Revenue Growth Potential: Initial annual revenues are modest but expected to grow at a CAGR of 15-20%, contingent on clinical validation, reimbursement, and market expansion.
• Competitive and Regulatory Risks: Evolving competition and regulatory requirements necessitate ongoing evidence generation and strategic positioning.
• Manufacturing and Cost Constraints: Efficient supply chain management and optimization are vital for profitability.
• Future Opportunities: Label expansion, combination therapies, and pipeline advancements underpin long-term financial prospects.

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FAQs

1. What are the primary clinical benefits of golodirsen for DMD patients?
Golodirsen induces exon 53 skipping, leading to increased production of functional dystrophin protein, which can slow disease progression and improve muscle function in eligible patients.

2. How does the small patient population affect golodirsen’s market potential?
The targeted patient subset limits total sales volume, emphasizing the importance of pricing strategies and reimbursement negotiations to optimize revenue within a constrained market.

3. What are the key regulatory challenges facing golodirsen’s further adoption?
Post-approval confirmatory studies are required due to initial accelerated approval status; failure to confirm efficacy could lead to restrictions or withdrawal.

4. How might emerging therapies impact golodirsen’s market?
Gene editing and other advanced modalities may offer more durable or broad-spectrum treatments, potentially challenging oligonucleotide-based therapies’ market share.

5. What strategies can Sarepta adopt to enhance golodirsen’s market penetration?
Enhancing clinical data, expanding indications, engaging payers with cost-effectiveness evidence, and streamlining manufacturing are critical strategies for growth.

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References

[1] Romitti, P. A., et al. (2015). Prevalence of Duchenne and Becker muscular dystrophies in the United States. Human Genetics, 134(12), 1275-1284.
[2] Aartsma-Rus, A., & Van Deutekom, J. C. (2020). Exon skipping in Duchenne muscular dystrophy: prospects and challenges. Current Opinion in Neurology, 33(5), 648-654.
[3] Biopharma. (2020). Oral presentation on casimersen's clinical data. Biopharmaceutical Reports.
[4] Sarepta Therapeutics. (2020). Annual Report.
[5] Sarepta Therapeutics. (2022). Q4 and full-year financial results.

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Disclaimer: The information provided is for informational purposes only and not investment advice.