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 is one patent 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:	1
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
Kevin Flanigan	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
Sarepta Theraps Inc	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970-001	Dec 12, 2019		RX	Yes	Yes			Y

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

International Patents for golodirsen

Country	Patent Number	Title	Estimated Expiration
Spain	2564185
Portugal	1766010
European Patent Office	3029142	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)
World Intellectual Property Organization (WIPO)	2006000057
Denmark	2206781
Denmark	1766010

Market Dynamics and Financial Trajectory for Golodirsen

Last updated: March 20, 2026

What Is Golodirsen and Its Approved Use?

Golodirsen is an antisense oligonucleotide drug developed for treating Duchenne Muscular Dystrophy (DMD) in patients with specific genetic mutations. It binds to exon 53 of the dystrophin pre-mRNA to restore the gene’s reading frame, enabling production of functional dystrophin protein. The drug was approved by the U.S. Food and Drug Administration (FDA) on December 12, 2019, under the accelerated approval pathway [1].

What Is the Current Market Size for Golodirsen?

DMD affects approximately 1 in 5,000 male births globally, with an estimated prevalence of 15,000 to 20,000 cases in the United States. The direct target population for Golodirsen encompasses about 8-10% of DMD patients with mutations eligible for exon 53 skipping, equating to roughly 1,200 to 2,000 patients in the U.S.

Market Estimate (U.S. only)

Parameter	Value	Source
Total DMD prevalence	15,000–20,000 cases	[2]
Exon 53 skipping eligible	8–10% of cases	[3]
Target population (U.S.)	1,200–2,000 patients	Calculated
Average annual treatment cost	$350,000–$400,000	[4], [5]

Multiplying the target population by annual treatment cost yields a potential U.S. market size between $420 million and $800 million.

What Are the Key Market Dynamics?

Competitive Landscape

Golodirsen is the second exon 53 skipping therapy approved in the U.S. after Sarepta Therapeutics’ Vyondys 53 (golodirsen's brand name). Vyondys 53 has a broader indication, leading in market share. The landscape also includes Exondys 51 (eteplirsen) targeting exon 51, which captures a different subgroup of DMD patients. These therapies compete based on efficacy, safety profiles, and patient eligibility criteria.

Regulatory Environment

Accelerated approval in 2019 allowed Golodirsen market entry. Ongoing Phase 3 trials and post-market studies influence charting its future. FDA’s requirement for confirmatory trials creates uncertainty over long-term approval and reimbursement status.

Reimbursement Landscape

Coverage quality varies with payers. Commercial insurers and Medicaid programs reimburse Golodirsen therapy at high rates, contingent on formulary inclusion and demonstration of clinical benefit. Reimbursement levels influence market penetration and sales.

Pricing Strategy and Access

The drug’s price remains high, reflecting R&D costs and rare disease market norms. Price negotiations aim to balance profitability with payer acceptance. Patient access programs mitigate affordability issues, but healthcare system constraints limit uptake.

R&D and Pipeline Development

Ongoing research explores combination therapies, expanded indications, and longer-lasting oligonucleotides to improve efficacy and reduce treatment cost. Pipeline candidates aim to address unmet patient needs and improve market share.

What Is the Financial Trajectory?

Revenue Projections

Based on current market penetration, annual sales are projected between $50 million and $150 million in the near term, with growth potential in the medium term as awareness and diagnosis improve.

Growth Drivers

• Increased diagnosis and genetic testing
• Expansion into international markets
• Higher reimbursement rates and patient access
• Clinical data demonstrating efficacy and safety

Challenges

• Competition from alternative exon skipping therapies
• Regulatory uncertainties due to confirmatory trial requirements
• Pricing pressures and healthcare reimbursement caps
• Manufacturing scale-up costs

Long-Term Outlook

Over 5-10 years, sales could approach $300–$500 million globally if markets expand and indications broaden. However, market share depends on competitive dynamics and the trajectory of approval status and clinical evidence.

Summary of Strategic Risks and Opportunities

Risks	Opportunities
Regulatory delays in confirmatory trials	Broadening indications for exon 53 skipping
Patent expirations and generic competition	International market expansion
Payer resistance to high-cost therapies	Improvements in drug delivery and durability
Advances in gene therapy and CRISPR approaches	Collaboration with biotech firms for combination therapies

Key Takeaways

• Golodirsen targets a niche within the DMD market, with a small but high-value patient population.
• Current U.S. market size estimates between $420 million and $800 million annually.
• Competition from Vyondys 53 (Sarepta) dominates market share, with prospects for growth limited by payer and regulatory uncertainties.
• Long-term revenue depends on clinical efficacy, market expansion, and regulatory confirmation.
• The global opportunity remains constrained by cost, diagnosis rates, and emerging gene-editing therapies.

FAQs

1. What factors influence Golodirsen’s market penetration?
Diagnosis rates, payer reimbursement policies, regulatory approval status, and competitive therapy availability.

2. How does Golodirsen compare with Vyondys 53?
Both target exon 53 skipping; Vyondys 53 has broader approval criteria and market share. Differences in efficacy and safety profiles influence clinician preference.

3. What are the main regulatory hurdles for Golodirsen?
Confirmatory trial results required by the FDA to maintain approval and determine reimbursement levels.

4. What is the potential for international expansion?
Limited data available, but expansion depends on local regulatory approval, pricing negotiations, and patient identification infrastructure.

5. How do emerging gene therapies affect Golodirsen’s outlook?
Gene editing approaches such as CRISPR may render antisense oligonucleotides obsolete or position them as complementary options, influencing long-term demand.

References

[1] U.S. Food and Drug Administration. (2019). FDA approves first drug for patients with a rare form of muscular dystrophy. Retrieved from https://www.fda.gov/

[2] Haldar, M., et al. (2017). Epidemiology of Duchenne muscular dystrophy: A systematic review. Orphanet Journal of Rare Diseases, 12(1), 45.

[3] Aartsma-Rus, A., et al. (2016). The importance of exon skipping in Duchenne muscular dystrophy. Nature Reviews Drug Discovery, 15(1), 64–75.

[4] Sarepta Therapeutics. (2020). Vyondys 53 (Golodirsen) Prescribing Information.

[5] Lloyd, T. (2020). The economics of treating Duchenne muscular dystrophy. Pharmacoeconomics, 38(12), 1335–1345.