Sarepta Therapeutics Boston Consulting Group Matrix
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Sarepta Therapeutics' position within the BCG Matrix is crucial for understanding its product portfolio's growth potential and market share. A quick glance reveals key areas of focus, but to truly grasp the strategic implications and future trajectory, a deeper dive is essential.
Uncover the full BCG Matrix for Sarepta Therapeutics and gain a clear view of where its products stand—Stars, Cash Cows, Dogs, or Question Marks. Purchase the full version for a complete breakdown and strategic insights you can act on.
Stars
Elevidys (delandistrogene moxeparvovec) - Ambulatory DMD
Elevidys, Sarepta Therapeutics' flagship gene therapy for ambulatory Duchenne muscular dystrophy (DMD), is a prime example of a company's potential star. Its revenue trajectory is impressive, with $281.9 million in U.S. net product revenue reported for the second quarter of 2025. This strong performance underscores its market leadership and significant contribution to Sarepta's overall financial health.
The expanded label approval in June 2024 was a pivotal moment, broadening Elevidys' reach to a larger patient population and reinforcing its dominant market share in the expanding DMD treatment landscape. While a brief shipment pause occurred in July 2025 due to safety reviews, the resumption of infusions for ambulatory patients signals robust demand and a clear path for continued growth.
Expanded Label & Global Approvals for Elevidys
Sarepta Therapeutics' Elevidys, a groundbreaking gene therapy for Duchenne muscular dystrophy (DMD), has seen its market potential significantly amplified. In June 2024, the U.S. Food and Drug Administration (FDA) expanded the therapy's label, making it accessible to over 80% of DMD patients in the United States who have a confirmed mutation in the DMD gene. This broadens the addressable patient population considerably, positioning Elevidys for substantial growth.
The global expansion of Elevidys is also accelerating. Following its U.S. approval, the therapy received a green light in Japan in May 2025. Additionally, the European Medicines Agency (EMA) is currently reviewing its marketing authorization application. These international approvals and ongoing reviews are crucial for Elevidys's trajectory as a high-growth product within Sarepta's portfolio.
Leading Position in DMD Gene Therapy
Sarepta Therapeutics is a true leader in Duchenne muscular dystrophy (DMD) gene therapy, largely thanks to Elevidys. It was the very first and remains the only approved gene therapy for this debilitating condition. This significant first-mover advantage has allowed Sarepta to capture a substantial share of the DMD treatment market, which is growing rapidly.
The company isn't resting on its laurels; it's actively investing in generating more clinical data. Positive results from studies like EMBARK and ENDEAVOR further solidify Elevidys' efficacy and safety profile. This commitment to data is crucial for maintaining their leading market share in such a high-growth therapeutic area.
High Revenue Contribution from Elevidys
Elevidys is the driving force behind Sarepta Therapeutics' impressive financial trajectory, with its sales acting as the primary catalyst for substantial revenue growth. The drug's commercial success is clearly reflected in the company's performance figures.
- Elevidys' Impact on Revenue: In the second quarter of 2025, Sarepta's total revenues saw a significant jump of $248.2 million compared to the same period in 2024. This surge was predominantly fueled by a $160.1 million increase in net product revenue specifically from Elevidys.
- Year-to-Date Performance: For the first six months of 2025, Sarepta's total net product revenue climbed to $1,355.9 million. This represents a considerable increase from $776.4 million in the corresponding period of the previous year, highlighting Elevidys' dominant contribution.
- Star Category Justification: The robust commercial adoption and resulting revenue generation firmly position Elevidys as a Star in Sarepta's portfolio. This strong performance indicates its high market share and growth potential, generating significant cash flow that can be reinvested into other areas of the business.
Continued Investment in Elevidys Commercialization
Sarepta Therapeutics is doubling down on Elevidys, its Duchenne muscular dystrophy gene therapy. This strategic investment in commercialization and further clinical trials aims to solidify Elevidys' position as a market leader. The company is actively addressing regulatory hurdles, particularly regarding safety labeling and risk mitigation for non-ambulatory patients, following a temporary shipment pause.
This proactive approach to expanding patient access and resolving FDA-related matters underscores Sarepta's commitment to maximizing Elevidys' market potential.
- Continued Investment: Sarepta is channeling significant resources into Elevidys' commercial rollout and ongoing clinical studies.
- Regulatory Engagement: The company is actively collaborating with the FDA on safety labeling and risk mitigation strategies for non-ambulatory patients.
- Market Potential: Addressing these challenges is crucial for unlocking Elevidys' full market potential and maintaining its leadership.
- Star Status Reinforcement: The sustained investment and focus on market expansion reinforce Elevidys' classification as a Star in the BCG matrix.
Elevidys: A $281.9M Star in Q2 2025!
Elevidys has firmly established itself as a Star within Sarepta Therapeutics' product portfolio. Its significant revenue contribution, driven by strong U.S. net product sales of $281.9 million in Q2 2025, highlights its market dominance. The expanded U.S. label approval in June 2024 broadened its patient reach, solidifying its position as the leading gene therapy for Duchenne muscular dystrophy.
The global expansion efforts, including approval in Japan in May 2025 and ongoing review by the EMA, further underscore Elevidys' high growth potential. Sarepta's continued investment in clinical data and commercialization reinforces its status as a Star, generating substantial cash flow for future innovation.
| Product | Category | 2024 Revenue (Est.) | 2025 Revenue (Q2) | Growth Potential |
| Elevidys | Star | $1.0 Billion+ | $281.9 Million | High |
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Cash Cows
Exondys 51 (eteplirsen)
Exondys 51 (eteplirsen) is a cornerstone of Sarepta Therapeutics, recognized as a mature product within their portfolio. As an established RNA-based exon-skipping therapy for Duchenne Muscular Dystrophy (DMD) patients eligible for exon 51 skipping, it has secured a solid and substantial market share.
This therapy consistently contributes to Sarepta's revenue stream. For the second quarter of 2024, Exondys 51 reported net product sales of $129.8 million, underscoring its dependable financial performance within the PMO franchise.
While its growth trajectory may not match newer gene therapies, Exondys 51's established market position and predictable revenue generation solidify its role as a reliable cash cow for the company.
Vyondys 53 (golodirsen)
Vyondys 53, a crucial RNA-based therapy for Duchenne Muscular Dystrophy (DMD) patients with exon 53 skipping mutations, functions as a Cash Cow for Sarepta Therapeutics. Its established market presence within its specific niche ensures a consistent and reliable stream of revenue, similar to Exondys 51.
In the second quarter of 2024, Vyondys 53 generated $31.6 million in net product sales. This mature product requires minimal aggressive marketing efforts, allowing it to yield significant profit margins and contribute substantially to the company's overall financial health.
Amondys 45 (casimersen)
Amondys 45 (casimersen) is a key player in Sarepta Therapeutics' portfolio, fitting squarely into the Cash Cow quadrant of the BCG Matrix. It targets a specific niche within Duchenne muscular dystrophy (DMD) patients, those with mutations responsive to exon 45 skipping, thereby rounding out Sarepta's approved exon-skipping treatments.
This therapy has established a dominant market share within its designated DMD patient subgroup. In the second quarter of 2024, Amondys 45 achieved net product sales of $77.4 million, clearly demonstrating its consistent ability to generate significant revenue for the company.
The combined financial contributions from Sarepta's exon-skipping therapies, including Amondys 45, create a robust and stable revenue stream. This financial stability is crucial, enabling Sarepta to allocate resources towards developing and investing in potentially higher-growth opportunities within its pipeline.
Stable Revenue from PMO Franchise
Sarepta Therapeutics' portfolio of RNA-based PMO therapies, including Exondys 51, Vyondys 53, and Amondys 45, forms a significant Cash Cow. These established products generated a substantial $231.3 million in net product revenue during the second quarter of 2025, demonstrating their consistent market performance. Crucially, the introduction of Elevidys has not materially affected the revenue generated by these PMO therapies, highlighting their enduring appeal and market position. This stable revenue stream is vital for Sarepta, as it provides the financial foundation to invest in the development of its promising new pipeline assets while ensuring the company remains profitable.
- Stable Revenue: The PMO franchise consistently contributes significant revenue.
- Q2 2025 Performance: RNA-based PMOs generated $231.3 million in net product revenue.
- Market Resilience: Launch of Elevidys has not impacted PMO revenue.
- Funding R&D: This revenue supports investment in new pipeline development.
Established Market Share in Exon-Skipping DMD
Sarepta Therapeutics' exon-skipping therapies have secured a dominant position within their targeted Duchenne muscular dystrophy (DMD) patient segments. These treatments are designed for specific genetic mutations, ensuring a consistent and identifiable patient pool. The company reported approximately $1.3 billion in net product revenue for the full year 2023, with its exon-skipping products being the primary drivers.
These therapies benefit from long-standing market presence and strong acceptance within the DMD community. This translates into reliable demand and predictable revenue generation for Sarepta. For instance, the U.S. Food and Drug Administration (FDA) approved Elevidys (delandistrogene moxeparvovec-usld) in June 2023, a gene therapy for ambulatory pediatric patients with Duchenne muscular dystrophy, marking a significant advancement in the field and potentially complementing their existing exon-skipping portfolio.
- Dominant Market Position: Sarepta's exon-skipping therapies hold substantial market share in their specific Duchenne muscular dystrophy (DMD) patient populations.
- Targeted Patient Base: The therapies address distinct genetic mutations, serving a well-defined and stable patient demographic.
- Revenue Stability: Their established presence and community acceptance ensure ongoing demand and predictable revenue streams.
- Cash Cow Status: This strong market share in a mature segment of the DMD market firmly establishes these therapies as valuable cash cows for Sarepta.
Sarepta's PMO Therapies: The Financial Backbone
Sarepta's established exon-skipping therapies, including Exondys 51, Vyondys 53, and Amondys 45, function as its primary cash cows. These mature products consistently generate substantial revenue by addressing specific genetic mutations in Duchenne Muscular Dystrophy patients.
Their strong market penetration and established patient base ensure a predictable and reliable income stream for Sarepta Therapeutics. This financial stability is crucial for funding ongoing research and development efforts, particularly for newer therapies like Elevidys.
In Q2 2024, these PMO therapies collectively generated $238.8 million in net product sales, underscoring their enduring financial contribution to the company.
| Product | Q2 2024 Net Sales (Millions USD) | BCG Matrix Category |
| Exondys 51 | $129.8 | Cash Cow |
| Vyondys 53 | $31.6 | Cash Cow |
| Amondys 45 | $77.4 | Cash Cow |
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Dogs
Discontinued PPMO Program (SRP-5051)
Sarepta Therapeutics discontinued its SRP-5051 program for Duchenne muscular dystrophy in November 2024. This decision stemmed from safety issues, FDA feedback, and the strong market performance of their existing therapy, Elevidys.
Programs like SRP-5051, which falter on efficacy or safety, or are outpaced by better treatments, are typically categorized as Dogs. These initiatives drain capital and resources without generating the expected returns.
Paused LGMD Gene Therapy Programs (excluding SRP-9003)
Sarepta Therapeutics, in a strategic pivot announced in July 2025, has paused the majority of its gene therapy programs targeting limb-girdle muscular dystrophy (LGMD). This move directly follows an FDA clinical hold initiated in July 2025, stemming from safety concerns, including reported patient fatalities potentially associated with the AAVrh74 vector.
While SRP-9003 for LGMD2E/R4 remains on track for a Biologics License Application (BLA) submission, the other paused LGMD gene therapy candidates are now viewed as having limited market share and uncertain future growth. These factors position them as Question Marks within Sarepta's current BCG Matrix, requiring careful evaluation of future investment versus potential divestment.
Revoked AAVrh74 Platform Technology Designation
In July 2025, the FDA revoked Sarepta Therapeutics' platform technology designation for its AAVrh74 vector. This decision followed the emergence of new safety data, including patient fatalities, which raised concerns about the platform's overall safety profile for broader applications.
The FDA's assessment indicated that the preliminary evidence was insufficient to support the platform's use across multiple drugs without significant adverse safety effects. This regulatory action directly impacts the perceived safety and future utility of therapies relying on the AAVrh74 vector, suggesting a diminished growth outlook for its general deployment.
Programs Divested or Shelved Post-Restructuring
Sarepta Therapeutics' strategic restructuring in July 2025 led to the divestment or shelving of certain programs. This move was part of a broader effort to concentrate resources on key therapeutic areas, specifically their siRNA platform.
The restructuring, which included a workforce reduction of about 500 employees, aimed to cut annual expenses by approximately $400 million starting in 2026. Programs no longer considered core to Sarepta's forward-looking strategy, or those being pursued for strategic alternatives like partnerships, fall into this category.
- Pipeline Reprioritization: Programs not aligned with the sharpened focus on high-impact areas like the siRNA platform were divested or shelved.
- Workforce Reduction: Approximately 500 employees were impacted as part of the restructuring to streamline operations.
- Expense Reduction Target: The company targeted an annual expense reduction of around $400 million, effective from 2026.
- Focus on Core Assets: This strategic shift aimed to enhance Sarepta's concentration on its most promising and advanced therapeutic candidates.
Therapies with Limited Patient Population and Stagnant Growth
Within Sarepta's portfolio, therapies targeting extremely rare genetic disorders, despite regulatory approval, can become Dogs if their patient populations remain critically small and show no discernible growth. These products would likely be older assets that have been superseded by newer, more effective treatments or those facing significant reimbursement challenges that limit access. For instance, a therapy approved for a condition affecting fewer than 100 individuals globally, with no pipeline expansion or new indications on the horizon, would fit this profile.
While Sarepta’s current approved Duchenne muscular dystrophy (DMD) therapies like Exon 51 (golodirsen) and Exon 53 (casimersen) are considered Cash Cows due to their established market presence and revenue generation, any prior or deprioritized assets would be candidates for the Dog category. These might include older gene therapy candidates that failed to meet efficacy endpoints in early trials or small molecule drugs that proved ineffective against specific mutations. Such products would see minimal to no further investment, with the strategy focused on managing existing obligations or planning for eventual discontinuation.
- Limited Patient Reach: Therapies for ultra-rare conditions with patient populations in the dozens, leading to minimal revenue.
- Stagnant Growth: Lack of new indications, clinical trial failures, or market competition hindering expansion.
- Deprioritized Assets: Older products or candidates that have been sidelined in favor of newer, more promising treatments.
- Loss Minimization Strategy: Focus on reducing operational costs and exploring divestiture rather than growth investment.
Sarepta's Pipeline: A BCG Matrix Dog's Tale
Sarepta’s discontinued SRP-5051 program for Duchenne muscular dystrophy, due to safety concerns and FDA feedback in November 2024, exemplifies a Dog in the BCG Matrix. Similarly, the majority of its LGMD gene therapy programs, paused in July 2025 following an FDA clinical hold due to safety issues, are now viewed as Dogs with limited market share and uncertain futures.
These programs, characterized by low market share and low growth potential, consume resources without generating significant returns. The revocation of Sarepta's AAVrh74 platform technology designation in July 2025 further solidifies the diminished outlook for related therapies, pushing them further into the Dog quadrant.
The strategic restructuring in July 2025, including a workforce reduction of approximately 500 employees and a target of $400 million in annual expense cuts from 2026, involved divesting or shelving programs not aligned with the company's core focus on its siRNA platform. These divested or shelved programs, unable to gain traction or facing insurmountable challenges, are prime examples of Sarepta's Dogs.
Therapies for ultra-rare disorders with minimal patient populations and no growth prospects, or older treatments superseded by newer, more effective options, also fall into the Dog category. These assets are managed to minimize losses rather than for growth.
| Program Status | BCG Category | Reasoning | Key Dates/Events | Financial Implication |
| SRP-5051 (DMD) | Dog | Discontinued due to safety issues and FDA feedback. | November 2024 | Resource drain, no expected returns. |
| LGMD Gene Therapies (Majority) | Dog | Paused due to FDA clinical hold (safety concerns) and diminished growth outlook. | July 2025 | Limited market share, uncertain future, capital consumption. |
| Divested/Shelved Programs | Dog | Reprioritized out of core strategy, facing divestment or discontinuation. | July 2025 Restructuring | Reduced operational expenses, focus on core assets. |
| Ultra-Rare Disorder Therapies (Stagnant) | Dog | Critically small patient populations, no discernible growth, or superseded by newer treatments. | Ongoing | Minimal revenue, focus on loss minimization. |
Question Marks
Elevidys (non-ambulatory DMD population)
Elevidys for the non-ambulatory Duchenne muscular dystrophy (DMD) population is currently a Question Mark in Sarepta Therapeutics' BCG Matrix. While the drug shows promise in a high-growth market, its current market share is limited due to ongoing regulatory discussions.
The FDA's temporary pause on Elevidys shipments in July 2025, though lifted for ambulatory patients, highlights the safety concerns that need to be addressed for the non-ambulatory segment. Discussions with the FDA are crucial for defining safety labeling and a risk-mitigation strategy, impacting market access.
This segment presents a significant growth opportunity if Sarepta can successfully navigate the regulatory landscape and demonstrate a favorable safety profile for non-ambulatory individuals. However, until these hurdles are cleared, its market share will remain constrained.
SRP-9003 (LGMD2E/R4 gene therapy)
SRP-9003, Sarepta's gene therapy for Limb-Girdle Muscular Dystrophy Type 2E/R4 (LGMD2E/R4), is a prime example of a Question Mark in their BCG matrix. The company aims to submit a Biologics License Application (BLA) for SRP-9003 in the latter half of 2025, indicating progress despite broader regulatory challenges.
This therapy targets a new, high-growth area for Sarepta, but it currently holds no market share. The FDA's previous clinical hold on other LGMD gene therapies and the revocation of the AAVrh74 platform designation have increased scrutiny on the program.
siRNA Program for Facioscapulohumeral Muscular Dystrophy (FSHD) - SRP-1001
Sarepta Therapeutics' SRP-1001 program targeting Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1) fits the profile of a Question Mark in the BCG Matrix. This early-stage asset is currently in Phase 1/2 clinical trials, with preliminary data anticipated in the latter half of 2025.
FSHD presents a substantial unmet medical need and a promising high-growth market for Sarepta beyond its existing Duchenne Muscular Dystrophy (DMD) franchise. However, as a nascent program, SRP-1001 currently holds no market share and necessitates significant capital investment for continued clinical development and regulatory navigation.
siRNA Program for Myotonic Dystrophy Type 1 (DM1) - SRP-1003
SRP-1003 represents Sarepta Therapeutics' siRNA program targeting Myotonic Dystrophy Type 1 (DM1). This rare neuromuscular disorder presents a substantial unmet medical need, positioning SRP-1003 as a high-potential asset within Sarepta's broader pipeline.
The DM1 program, similar to Sarepta's focus on FSHD, targets a rare disease market with significant growth potential for a breakthrough therapy. The company anticipates initial Phase 1/2 trial data in the latter half of 2025.
- Program: SRP-1003 (siRNA for Myotonic Dystrophy Type 1)
- Market: Rare neuromuscular disorder with high unmet need
- Clinical Timeline: Phase 1/2 data expected H2 2025; potential launch ~2029
- BCG Classification: Potential Star, requiring significant R&D investment
Other Early-Stage Pipeline Assets
Sarepta Therapeutics’ early-stage pipeline extends beyond its core Duchenne muscular dystrophy programs. The company is actively developing other small interfering RNA (siRNA) therapies, such as SRP-1004 targeting Spinocerebellar Ataxia Type 2 (SCA2) and SRP-1005 for Huntington's Disease. These initiatives, alongside early gene editing programs for Duchenne, represent significant future growth opportunities.
These nascent programs are currently in the discovery or initial clinical trial stages, focusing on neurological disorders and rare muscle-debilitating conditions with significant unmet medical needs. While they hold the promise of substantial long-term expansion and diversification for Sarepta, they are characterized by no current market presence and demand considerable, high-risk investment in research and development.
- SRP-1004 (SCA2): Early-stage development, potential for neurological disease treatment.
- SRP-1005 (Huntington's Disease): siRNA program in preclinical or early clinical phases.
- Gene Editing for Duchenne: Nascent programs exploring novel therapeutic modalities.
- High-Risk, High-Reward: These assets require significant R&D investment with uncertain outcomes, reflecting their position as potential future Stars or current Dogs in the BCG matrix.
Future of Sarepta's Pipeline: Uncertain?
Sarepta's gene therapy for Limb-Girdle Muscular Dystrophy Type 2E/R4 (LGMD2E/R4), SRP-9003, is a Question Mark. The company plans to submit a Biologics License Application (BLA) in the latter half of 2025, targeting a high-growth area with no current market share.
SRP-1001, targeting Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1), is also a Question Mark. It's in early-stage trials with preliminary data expected in the second half of 2025, representing a significant market opportunity but requiring substantial investment.
SRP-1003, an siRNA therapy for Myotonic Dystrophy Type 1 (DM1), is another Question Mark. Initial Phase 1/2 trial data is anticipated in the latter half of 2025 for this rare disease with high unmet need.
Other early-stage programs like SRP-1004 (SCA2) and SRP-1005 (Huntington's Disease) are also Question Marks. These nascent therapies focus on neurological disorders and require considerable, high-risk investment with uncertain outcomes.
| Program | Target Indication | BCG Status | Market Potential | Current Market Share | Key Developments (2025) |
|---|---|---|---|---|---|
| Elevidys (non-ambulatory) | Duchenne Muscular Dystrophy | Question Mark | High Growth | Limited | Ongoing regulatory discussions for safety labeling |
| SRP-9003 | LGMD2E/R4 | Question Mark | High Growth | None | BLA submission planned H2 2025 |
| SRP-1001 | FSHD1 | Question Mark | High Growth | None | Phase 1/2 data anticipated H2 2025 |
| SRP-1003 | Myotonic Dystrophy Type 1 (DM1) | Question Mark | High Growth | None | Phase 1/2 data anticipated H2 2025 |
BCG Matrix Data Sources
Our BCG Matrix leverages Sarepta's financial disclosures, industry growth projections, and product pipeline analysis to accurately position each therapeutic area.
