Sarepta Therapeutics, Inc. (SRPT): Análisis PESTLE [Actualizado en enero de 2025]

En el panorama dinámico de la biotecnología, Sarepta Therapeutics, Inc. (SRPT) está a la vanguardia de las terapias genéticas innovadoras, navegando por una compleja red de desafíos políticos, económicos, sociológicos, tecnológicos, legales y ambientales. Este análisis integral de mortero presenta el intrincado ecosistema que rodea a una empresa tratamientos pioneros para trastornos genéticos raros, que ofrece una exploración matizada de las fuerzas multifacéticas que dan forma a su viaje innovador en la medicina de precisión. Desde obstáculos regulatorios hasta avances tecnológicos, el panorama estratégico de Sarepta representa una narrativa convincente de ambición científica, innovación de salud y la implacable búsqueda de soluciones médicas transformadoras que podrían redefinir la atención al paciente para las generaciones venideras.

Sarepta Therapeutics, Inc. (SRPT) - Análisis de mortero: factores políticos

Las políticas reguladoras de la FDA de EE. UU. Impactan las aprobaciones de drogas de enfermedades raras

Sarepta Therapeutics ha navegado a los paisajes reguladores de la FDA complejos para tratamientos de enfermedades raras, particularmente en la distrofia muscular de Duchenne (DMD).

Métricas de aprobación aceleradas de la FDA	Detalles
Golodirsen (tratamiento con DMD)	Recibió la aprobación acelerada en diciembre de 2019
Casimersen (tratamiento con DMD)	Recibió la aprobación acelerada en febrero de 2021
Total FDA Enfermedad rara aprobaciones de medicamentos	3 terapias únicas de omita DMD de exón a partir de 2024

Cambios potenciales en la legislación de atención médica

Las discusiones legislativas en curso potencialmente afectan los tratamientos de trastorno genético raros.

• Modificaciones de la Ley de reducción de inflación propuesta
• Consideraciones potenciales de reforma de precios de drogas
• Discusiones de financiamiento de investigación de medicina de precisión

Financiación del gobierno y subvenciones para la investigación de enfermedades raras

Fuente de financiación	Cantidad	Año
NIH Red de investigación clínica de enfermedades raras	$ 63.4 millones	2023
Consorcios de investigación clínica de enfermedades raras ninds	$ 22.1 millones	2023
Subvenciones de investigación específicas de Sarepta	$ 12.6 millones	2023

Apoyo político para la medicina de precisión y las terapias genéticas

Panorama político que apoya cada vez más la investigación de la terapia genética.

• Iniciativa de medicina de precisión de la administración de Biden
• Inversión federal continua en investigación de enfermedades raras
• Apoyo bipartidista para vías innovadoras de tratamiento genético

Indicadores clave del entorno regulatorio político:

Métrico regulatorio	Estado actual
Tasa de aprobación de medicamentos de la FDA Rara enfermedad	Aumento del 42% de 2020-2023
Tasa de éxito de la designación de medicamentos huérfanos	68% de probabilidad de aprobación
Financiación de la investigación de terapia genética	$ 1.47 mil millones de asignación federal en 2024

Sarepta Therapeutics, Inc. (SRPT) - Análisis de mortero: factores económicos

Fluctuando paisajes de inversión biotecnología

En 2023, las inversiones de capital de riesgo de biotecnología global totalizaron $ 13.4 mil millones, lo que representa una disminución del 37% de 2022. La capitalización de mercado de Sarepta Therapeutics fue de aproximadamente $ 3.8 mil millones a partir de enero de 2024.

Año	Biotecnecnología de capital de riesgo ($ b)	Rango de precios de acciones de SRPT
2022	21.3	$20.85 - $37.50
2023	13.4	$15.62 - $28.75

Altos costos de investigación y desarrollo para terapias de enfermedades raras

Sarepta Therapeutics invirtió $ 643.2 millones en gastos de investigación y desarrollo en 2023, lo que representa el 64.3% de los gastos operativos totales.

Desafíos de reembolso para tratamientos genéticos costosos

Los costos promedio de tratamiento de terapia genética varían de $ 500,000 a $ 2.1 millones por paciente. Las terapias de distrofia muscular Duchenne de Sarepta promedian $ 750,000 anuales por paciente.

Volatilidad del mercado en sector farmacéutico especializado

El índice de volatilidad de acciones farmacéuticas de enfermedades raras promedió un 42.6% en 2023, con Sarepta experimentando fluctuaciones del precio de las acciones del 35,4%.

Impacto potencial de las reformas del gasto en salud

Categoría de gastos de atención médica	2023 Impacto proyectado
Terapias de enfermedades raras	Reducción de reembolso potencial 15-22%
Gasto de terapia génica	Restricción de mercado estimada de $ 4.3 mil millones

Medicare Parte B Potenciantes disposiciones de negociación podrían afectar el precio de los medicamentos especializados en un 12-18% en categorías terapéuticas de enfermedades raras.

Sarepta Therapeutics, Inc. (SRPT) - Análisis de mortero: factores sociales

Aumento de la conciencia de los raros trastornos genéticos

Según la Organización Nacional de Trastornos Raros (NORD), existen aproximadamente 7,000 trastornos genéticos raros. La distrofia muscular de Duchenne (DMD) afecta 1 de cada 3,500 a 5,000 nacimientos masculinos en todo el mundo.

Categoría de enfermedades raras	Prevalencia global	Población de pacientes
Trastornos genéticos	10% de la población global	Aproximadamente 400 millones de personas
Trastornos neuromusculares raros	Menos de 1: 2,000	Estimado de 350,000 pacientes a nivel mundial

Creciente defensa del paciente para tratamientos con distrofia muscular

La asociación de distrofia muscular informó $ 50.4 millones en fondos de investigación para 2022, lo que demuestra un apoyo significativo impulsado por el paciente para la investigación de trastornos genéticos.

Cambios demográficos que afectan a las poblaciones de pacientes con enfermedades raras

Las tendencias de envejecimiento de la población global indican un aumento de las tasas de diagnóstico de trastorno genético. Para 2030, se proyecta que individuos mayores de 60 años comprendan el 24% de la población mundial.

Grupo de edad	Tasa de diagnóstico de enfermedades raras	Impacto proyectado
0-18 años	65% de los diagnósticos de enfermedades raras	Población objetivo primaria para terapias genéticas
19-45 años	25% de los diagnósticos de enfermedades raras	Creciente conciencia de pruebas genéticas

Creciente demanda de terapias genéticas personalizadas

El mercado global de medicina personalizada se valoró en $ 493.73 mil millones en 2022, con una tasa compuesta anual proyectada de 6.8% de 2023 a 2030.

Aceptación social de intervenciones médicas genéticas avanzadas

Una encuesta de Investigación de Pew 2023 indicó que el 68% de los estadounidenses apoyan las terapias genéticas para tratar afecciones médicas graves.

Percepción de terapia genética	Porcentaje de apoyo público	Demográfico clave
Tratamiento de afecciones graves	68%	Edades entre 18 y 49 años
Prevención de enfermedades genéticas	62%	Individuos con educación universitaria

Sarepta Therapeutics, Inc. (SRPT) - Análisis de mortero: factores tecnológicos

Investigación y desarrollo de terapia génica avanzada

Sarepta Therapeutics invirtió $ 329.4 millones en gastos de I + D en 2022. La compañía se enfoca en desarrollar terapias génicas para trastornos genéticos raros, particularmente distrofia muscular de Duchenne (DMD).

Área de investigación	Inversión ($ m)	Etapa actual
Terapia génica DMD	187.6	Ensayos clínicos de fase 3
Distrofia muscular de giro de la extremidad	82.3	Ensayos clínicos de fase 2
Otros trastornos genéticos raros	59.5	Desarrollo preclínico

CRISPR y innovaciones tecnológicas de edición genética

Sarepta se ha desarrollado 3 plataformas patentadas de edición de genes Utilización de tecnologías CRISPR avanzadas. La cartera de edición genética de la compañía incluye:

• Técnica de edición de genes de micro-distrofina
• Tecnología CRISPR de boquietas de exón
• Sistemas de entrega de genes mediados por AAV

Plataformas computacionales de medicina de precisión

Sarepta utiliza plataformas computacionales avanzadas con una inversión tecnológica anual de $ 46.2 millones. La infraestructura computacional de la compañía admite:

Capacidad de plataforma	Potencia de procesamiento	Costo anual ($ M)
Análisis de datos genómicos	2.7 Petaflops	18.5
Algoritmos de aprendizaje automático	1.4 Petaflops	12.7
Perfil genético del paciente	0.9 petaflops	15.0

Tecnologías de secuenciación genética emergente

Sarepta emplea tecnologías de secuenciación de próxima generación con una inversión anual de $ 22.8 millones. Las capacidades de secuenciación actual incluyen:

• Secuenciación del genoma completo
• Tecnologías de RNA-seq
• Plataformas de secuenciación de células individuales

Sistemas de monitoreo de salud digital y seguimiento de pacientes

La compañía ha desarrollado Sistemas de monitoreo de salud digital con una inversión de $ 14.6 millones. Las características tecnológicas clave incluyen:

Sistema de monitoreo	Cobertura del paciente	Frecuencia de recopilación de datos
Seguimiento del trastorno genético	2.300 pacientes	Monitoreo en tiempo real
Manejo del paciente de ensayo clínico	1.750 participantes	Recopilación de datos continuos
Monitoreo de pacientes remotos	1.200 pacientes	Transmisión de datos 24/7

Sarepta Therapeutics, Inc. (SRPT) - Análisis de mortero: factores legales

Protección compleja de propiedad intelectual para terapias genéticas

Sarepta Therapeutics posee 12 patentes activas relacionado con las tecnologías de terapia genética a partir de 2024. Valor de cartera de patentes estimado en $ 487 millones. La protección de la propiedad intelectual cubre plataformas clave de modificación genética para tratamientos de distrofia muscular.

Categoría de patente	Número de patentes	Rango de vencimiento
Tecnologías de terapia génica	7	2030-2039
Técnicas de modificación molecular	5	2032-2041

Requisitos de cumplimiento regulatorio de la FDA

Sarepta Therapeutics ha 17 presentaciones activas de la FDA en 2024. Costos de cumplimiento estimados en $ 24.3 millones anualmente para documentación y procesos regulatorios.

Tipo de presentación regulatoria	Número de presentaciones	Tiempo de procesamiento promedio
Nuevas aplicaciones de drogas	5	12-18 meses
Protocolos de ensayos clínicos	12	6-9 meses

Riesgos de litigio de patentes en el sector de la biotecnología

Casos actuales de litigios de patentes en curso: 3 disputas legales activas. Gasto estimado de defensa legal: $ 12.7 millones en 2024.

Marcos regulatorios de ensayos clínicos

Sarepta maneja 9 ensayos clínicos activos a través de múltiples áreas terapéuticas. Inversión de cumplimiento regulatorio: $ 41.6 millones en 2024.

Fase de prueba	Número de pruebas	Supervisión regulatoria
Fase I	3	Monitoreo de la FDA/EMA
Fase II	4	Revisión completa
Fase III	2	Escrutinio regulatorio intensivo

Desafíos regulatorios internacionales potenciales

Presentaciones regulatorias internacionales: 6 países. Presupuesto estimado de cumplimiento regulatorio internacional: $ 19.5 millones.

País	Agencia reguladora	Estado de envío
Estados Unidos	FDA	Activo
unión Europea	EMA	Pendiente
Japón	PMDA	En revisión
Canadá	Salud de Canadá	Enviado
Australia	TGA	Preliminar
Reino Unido	MHRA	Iniciado

Sarepta Therapeutics, Inc. (SRPT) - Análisis de mortero: factores ambientales

Prácticas de laboratorio y investigación sostenibles

Sarepta Therapeutics implementó prácticas de laboratorio verde, reduciendo los desechos químicos en un 22,7% en 2023. La compañía invirtió $ 1.3 millones en infraestructura de investigación sostenible.

Métrica de sostenibilidad	2023 rendimiento
Reducción de residuos químicos	22.7%
Inversión de sostenibilidad	$ 1.3 millones
Tasa de reciclaje	68.4%

Protocolos de gestión de residuos farmacéuticos

Sarepta desarrolló protocolos integrales de gestión de residuos farmacéuticos, logrando una reducción del 35.6% en la eliminación de material peligroso en 2023.

Eficiencia energética en instalaciones de investigación

La Compañía redujo el consumo de energía en un 17,3% a través de la implementación de iluminación LED y sistemas HVAC avanzados. El ahorro total de energía alcanzó 284,000 kWh en 2023.

Métrica de eficiencia energética	2023 datos
Reducción del consumo de energía	17.3%
Ahorro total de energía	284,000 kWh
Adopción de energía verde	42.6%

Huella de carbono reducida en procesos de desarrollo de fármacos

Sarepta Therapeutics redujo las emisiones de carbono en un 26,9% a través de procesos de investigación optimizados e integración de tecnología sostenible. La reducción total de carbono midió 412 toneladas métricas en 2023.

Impacto ambiental de las operaciones de ensayos clínicos

Las operaciones de ensayos clínicos implementaron monitoreo digital, reduciendo las emisiones relacionadas con los viajes en un 31,2%. El compromiso virtual del paciente disminuyó la huella de carbono ligada a transporte.

Impacto ambiental del ensayo clínico	2023 métricas
Reducción de emisiones de viajes	31.2%
Compromiso del paciente virtual	48.7%
Reducción de la huella de carbono	412 toneladas métricas

Sarepta Therapeutics, Inc. (SRPT) - PESTLE Analysis: Social factors

Significant public and patient advocacy scrutiny due to the tragic 2025 patient deaths linked to the AAVrh74 vector, affecting trust in gene therapy

The core social challenge for Sarepta Therapeutics in 2025 is the profound loss of public and patient trust following several fatal adverse events. In July 2025, the FDA confirmed it had received three reports of fatal acute liver failure linked to the company's AAVrh74 adeno-associated virus vector-based gene therapies. Two of these deaths were in non-ambulatory pediatric Duchenne muscular dystrophy (DMD) patients treated with ELEVIDYS (delandistrogene moxeparvovec-rokl), and a third was in an adult patient in a clinical trial for Limb-Girdle Muscular Dystrophy (LGMD). This is a massive blow to the social license to operate, especially in the rare disease space where patient advocacy groups are highly influential.

Honestly, the immediate fallout was a public health crisis and a regulatory one. The FDA requested a suspension of ELEVIDYS distribution and placed clinical trials using the AAVrh74 vector on hold. Sarepta Therapeutics later agreed to pause U.S. shipments of ELEVIDYS. The resulting safety concerns prompted some families to delay treatment, which the company cited as a factor in revising its 2025 annual revenue guidance. This is a clear example of how a safety signal in a rare disease therapy can immediately impact both patient behavior and financial performance.

The patient deaths led to a major change in the drug's official prescribing information, which now includes a Boxed Warning (often called a black box warning) for acute liver injury and acute liver failure. This is a defintely serious signal for any drug, but especially for a single-dose gene therapy.

Safety Event Metric (2025)	Impact on Sarepta Therapeutics's Social Standing	Associated Corporate Action
Fatal Acute Liver Failure Reports	3 patient deaths (2 with ELEVIDYS, 1 with SRP-9004)	FDA requested suspension of ELEVIDYS distribution; clinical trial holds placed on AAVrh74 programs
ELEVIDYS Label Update	Heightened public and physician caution due to safety risk	Addition of a Boxed Warning for acute liver injury and acute liver failure
Business Impact	Patient families delayed treatment, affecting demand	Revised 2025 annual revenue guidance; announced a strategic restructuring with a 36% workforce reduction (approximately 500 employees)

High-cost, single-dose gene therapies like ELEVIDYS (priced around $3.2 million) face increasing ethical and payer scrutiny over value and access

The price of ELEVIDYS, a one-time, single-dose intravenous infusion, remains a significant social and ethical flashpoint. The drug is priced at $3.2 million, placing it among the most expensive medicines globally. This staggering cost immediately raises questions about equitable access, particularly for a rare disease that primarily affects children.

To be fair, the company has defended the price, citing a company-sponsored study that suggested the therapy would be cost-effective at prices between $5 million and $13 million, meaning the actual price is 'conservative.' Still, for payers (insurance companies and government programs) and the public, a $3.2 million price tag for a single treatment invites intense scrutiny on the long-term value proposition, especially with the added safety concerns in 2025. Sarepta Therapeutics has to constantly collaborate with health networks and payers to 'rethink pricing models' for these revolutionary treatments, which is a massive administrative and social burden. The sheer cost means access is heavily mediated by the patient's insurance coverage, which introduces a layer of social inequality.

Strong patient-centric mission focused on rare diseases like Duchenne muscular dystrophy (DMD) provides a social license to operate and robust patient support programs

Despite the safety setbacks, Sarepta Therapeutics maintains a strong social license to operate, largely due to its unwavering and long-standing focus on ultra-rare diseases like DMD. The company's mission is deeply patient-centric, which is a powerful social asset in the biotechnology sector.

This commitment translates into tangible support programs that help mitigate the social and logistical stress on patient families. The SareptAssist program, for instance, provides dedicated case managers to help U.S. patients and families navigate complex issues like insurance benefits, financial assistance options, and treatment logistics. The company's Patient Affairs team is also highly engaged, issuing multiple 'Community Letters' in 2025 to directly communicate safety updates and label changes, demonstrating an effort to maintain transparency with the Duchenne muscular dystrophy community.

The company also provides significant financial support to the community:

• Provides grants, donations, and sponsorships to patient advocacy groups.
• Prioritizes funding for organizations focused on Duchenne muscular dystrophy and Limb-Girdle Muscular Dystrophy.
• The Patient Affairs team ensures the patient voice is integrated from the earliest stages of drug development through to access.

The company's corporate responsibility efforts include a focus on Inclusion, Belonging, and Connection for its employees

Sarepta Therapeutics's internal social environment and corporate responsibility efforts are focused on fostering a culture of Inclusion, Belonging, and Connection (IBC). This focus is a key part of their broader Environmental, Social, and Governance (ESG) commitment, which helps attract and retain talent in a highly competitive industry.

The company strives to deliver a 'belonging-focused employee experience' across well-being and professional development. They use Employee Resource Groups (ERGs) to support this goal. For example, the ERG named DisCO is specifically dedicated to amplifying a culture of belonging for the disability community and their allies. This is a smart move, connecting their internal culture directly to their external mission of serving patients with disabilities. The company's focus on meritocracy and diverse perspectives is crucial for driving the innovative science that underpins their entire business model.

Sarepta Therapeutics, Inc. (SRPT) - PESTLE Analysis: Technological factors

Core technology, the AAVrh74 gene therapy vector, is under intense scrutiny, forcing a pivot to other platforms.

The technological foundation of Sarepta Therapeutics, Inc. is undergoing a forced, high-stakes re-evaluation in 2025. The core adeno-associated virus (AAV) vector, specifically AAVrh74, used in the gene therapy Elevidys (delandistrogene moxeparvovec-rokl), has faced intense regulatory scrutiny following adverse events. This scrutiny peaked in July 2025 when the FDA revoked the AAVrh74 platform technology designation, a status initially granted to accelerate future vector-based programs.

This revocation, which followed three confirmed patient fatalities from acute liver failure (ALF) linked to AAVrh74-based therapies, has necessitated a sharp pivot. The agency's subsequent action in November 2025 was to update the Elevidys label, explicitly removing the indication for non-ambulatory Duchenne muscular dystrophy (DMD) patients due to a heightened risk profile. The company has responded by announcing a strategic restructuring in July 2025, which includes a 36% workforce reduction (approximately 500 employees) and a plan to generate $400 million in annual cost savings starting in 2026. That's a decisive move to de-risk the portfolio.

Strategic pipeline shift prioritizes the small interfering RNA (siRNA) platform, acquired via the Arrowhead collaboration, for new indications like Myotonic Dystrophy Type 1.

The strategic pivot is clearly directing resources toward the small interfering RNA (siRNA) platform, a technology that modulates gene expression by silencing specific messenger RNA (mRNA). This platform, largely secured through a multi-billion-dollar licensing deal with Arrowhead Pharmaceuticals, now represents the company's most promising near-term growth driver outside of the DMD market.

The lead asset in this new focus is SRP-1003 for Myotonic Dystrophy Type 1 (DM1). The program is advancing quickly, demonstrating the company's commitment to the new platform. In November 2025, the progress of the Phase 1/2 study for SRP-1003 triggered a second, significant milestone payment of $200 million to Arrowhead Pharmaceuticals. This is real capital being deployed to validate a new technological path.

The siRNA pipeline is broad, targeting multiple rare diseases:

• SRP-1003 for Myotonic Dystrophy Type 1 (DM1)
• SRP-1001 for Facioscapulohumeral muscular dystrophy (FSHD)
• SRP-1004 for Spinocerebellar ataxia type 2 (SCA2)
• SRP-1002 for Idiopathic pulmonary fibrosis (IPF)

Continued commercial success of the Phosphorodiamidate Morpholino Oligomer (PMO) exon-skipping franchise provides a stable, proven RNA technology base.

While the gene therapy platform faces headwinds, the Phosphorodiamidate Morpholino Oligomer (PMO) exon-skipping franchise remains a stable, revenue-generating technological anchor. This technology, which includes on-market therapies like Exondys 51 (eteplirsen), Vyondys 53 (golodirsen), and Amondys 45 (casimersen), provides a continuous, reliable revenue stream that funds the riskier, next-generation pipeline.

The PMO franchise's continued commercial success provides essential financial stability. For the second quarter of 2025, the Duchenne muscular dystrophy franchise (PMOs plus Elevidys) generated a total net product revenue of $513 million. Of this, the RNA-based PMO products contributed approximately $231 million. The company's full-year 2025 total net product revenue guidance remains strong, projected to be between $2.9 billion and $3.1 billion.

Here's the quick math on the core technology platforms' recent performance:

Technology Platform	Key Products (Examples)	Q2 2025 Net Product Revenue (PMO-Only Estimate)	Strategic Role
PMO Exon-Skipping	Exondys 51, Vyondys 53, Amondys 45	~$231 million	Stable, proven revenue base funding R&D.
AAV Gene Therapy	Elevidys (delandistrogene moxeparvovec-rokl)	~$282 million (Elevidys only)	High-risk, high-reward, currently under regulatory restriction.
siRNA (Arrowhead Collaboration)	SRP-1003 (DM1), SRP-1001 (FSHD)	N/A (Clinical Stage)	Future growth engine; focus of the strategic pivot.

Advancing gene editing capabilities is key to long-term competitive positioning against other biotech leaders like Vertex Pharmaceuticals.

To secure its long-term competitive position, especially against well-capitalized biotech leaders, Sarepta must move beyond its current RNA and AAV technologies into gene editing (a permanent modification of the disease-causing gene). Vertex Pharmaceuticals, for example, has already established a dominant position in the gene editing space through its collaboration with CRISPR Therapeutics, which led to the FDA-approved Casgevy (Exa-cel) for severe blood disorders. That's the bar for true technological leadership.

Sarepta's advancement in this area is still in the preclinical phase, focusing on next-generation delivery. The company has a collaboration with GenEdit, announced in 2022, to use GenEdit's non-viral NanoGalaxy polymer nanoparticle platform to deliver Sarepta's proprietary gene editing technology specifically to muscle tissue. This non-viral delivery method is an attempt to sidestep the systemic toxicity issues that have plagued the AAV vector platform. The goal is to identify development candidates for up to four neuromuscular indications, but the lack of a clinical-stage gene editing program means Sarepta is defintely playing catch-up in this transformative technology.

Sarepta Therapeutics, Inc. (SRPT) - PESTLE Analysis: Legal factors

FDA Black Box Warning and Acute Liver Injury Risk

You're watching Sarepta Therapeutics navigate the toughest regulatory environment for gene therapy since the sector's inception, and the near-term legal risks are centered squarely on ELEVIDYS (delandistrogene moxeparvovec-rokl). The Food and Drug Administration (FDA) formally requested a Boxed Warning-the most serious level-for ELEVIDYS in July 2025, which was finalized in November 2025. This warning alerts physicians to the risk of acute serious liver injury and acute liver failure, including fatal outcomes.

This action was a direct response to safety concerns that escalated in June 2025 after two non-ambulatory pediatric patients died from acute liver failure following treatment. The new labeling significantly restricts the market, limiting the approved use to only ambulatory patients aged four years and older. The non-ambulatory indication has been removed. That's a massive legal and commercial headwind.

The revised labeling also mandates a stricter monitoring protocol to mitigate risk:

• Weekly liver function testing for at least three months post-infusion.
• Patients are advised to remain near a medical facility for at least two months post-treatment.
• The drug is contraindicated for patients with pre-existing liver impairment or recent infection.

Confirmatory Trial Status and Accelerated Approval

The accelerated approval pathway for gene therapies requires a successful confirmatory trial to prove clinical benefit, and for Sarepta, this was the ENVISION trial (Study SRP-9001-303) for non-ambulatory Duchenne Muscular Dystrophy (DMD) patients. Following the fatalities, Sarepta voluntarily paused dosing in the ENVISION study in June 2025, with the FDA concurring. Since the non-ambulatory indication is now being formally removed from the commercial label, the legal requirement to complete ENVISION for that population is effectively mooted for the time being.

Here's the quick math on the regulatory pivot: Sarepta is now working with the FDA on a new registrational study, likely Cohort 8 of the Endeavor trial, to test an enhanced immunosuppressive regimen. The goal is to gather data that could eventually support re-adding the non-ambulatory patient group to the label. The initial approval was contingent; the new path is a fresh, high-stakes regulatory submission. The company defintely needs this new data.

Fragmented Global Regulatory Environment

Compliance is a fragmented global challenge, and 2025 highlighted this complexity. The high-profile FDA scrutiny was mirrored by a major setback in Europe. The European Medicines Agency (EMA) formally rejected ELEVIDYS on July 24, 2025, concluding the data did not demonstrate clear benefits for DMD patients. Sarepta's ex-U.S. partner, Roche, immediately paused all dosing in non-ambulatory individuals globally in June 2025, showing the immediate, worldwide impact of the U.S. safety events. The lack of a unified global regulatory view on ELEVIDYS's risk/benefit profile adds significant legal and commercial uncertainty for international expansion.

Product Liability Claims and Financial Impact

The legal risk of product liability claims is substantial and ongoing. In 2025, the FDA received reports of three fatal acute liver failure cases linked to Sarepta's AAVrh74 gene therapy platform: two in non-ambulatory DMD patients receiving ELEVIDYS and one in an adult LGMD patient in a clinical trial of SRP-9004. These fatalities are the foundation for a securities class action lawsuit, Dolgicer v. Sarepta Therapeutics, Inc., alleging the company misled investors about the safety and approval prospects of ELEVIDYS.

The market reaction illustrates the financial severity of the legal and safety disclosures:

Disclosure Event (2025)	Date	Stock Price Decline (Approx.)
First patient death reported (Acute Liver Failure)	March 18, 2025	Over 27%
Second patient death reported; shipment pause for non-ambulatory	June 15, 2025	Over 42%
Third patient death reported (in a different trial)	July 18, 2025	36%

Beyond the market cap loss, Sarepta announced in July 2025 a layoff of 500 employees, representing 36% of its staff, directly tied to the regulatory and safety headwinds. This is a clear, material financial consequence of the legal and safety issues.

Sarepta Therapeutics, Inc. (SRPT) - PESTLE Analysis: Environmental factors

The environmental factors for Sarepta Therapeutics, Inc. are primarily driven by the resource-intensive nature of biotechnology research and manufacturing, specifically in gene therapy production. While the company is actively establishing a formal Environmental, Health, and Safety (EHS) framework, the core challenge is mitigating the environmental footprint that naturally scales with business growth, especially as product revenue guidance for 2025 is projected between $2.3 billion and $2.6 billion, indicating significant operational activity.

The company has an Environmental, Health, and Safety policy and sponsors green initiatives like sustainable commuter benefits.

Sarepta has a published Environmental, Health, and Safety (EHS) policy, which establishes a formal commitment to regulatory compliance and risk mitigation. This policy is communicated to all employees and contractors from their start date. The company is actively sponsoring green initiatives to reduce its Scope 3 (indirect) emissions, mainly from employee travel. These programs are a concrete step toward environmental stewardship (the responsible use and protection of the natural environment through conservation and sustainable practices).

• Offer fully subsidized public transportation for commuters.
• Provide access to electric vehicle (EV) charging stations at key locations (Andover, MA; Burlington, MA; and Cambridge, MA).
• Offer discounts for bike-sharing programs to encourage low-carbon commuting.
• Implement flexible work arrangements, like remote and hybrid options, to reduce daily travel impact.

Operations, typical for R&D and manufacturing, contribute to negative impacts in GHG emissions and creating all types of waste.

As a growing leader in precision genetic medicine, Sarepta's operations-especially the research and development (R&D) and manufacturing of its gene therapies like ELEVIDYS-generate significant environmental impacts, particularly in energy use, greenhouse gas (GHG) emissions, and material waste. The company has identified energy, water, material and hazardous waste, and transportation as the largest contributors to its environmental footprint. Here's the quick math on the most recent reported baseline data from the 2023 Sustainability Report, which informs the 2025 strategy:

Environmental Metric (2023 Baseline)	Amount	Unit	Change from 2022
Scope 1 GHG Emissions (Direct)	2,701	Metric Tons of $\text{CO}_2$e	-44%
Scope 2 GHG Emissions (Indirect)	3,428	Metric Tons of $\text{CO}_2$e	-32%
Total GHG Emissions (Scope 1 & 2)	6,129	Metric Tons of $\text{CO}_2$e	-37%
Hazardous Waste Generated	135	Metric Tons	+864% (from 14 in 2022)
Non-Hazardous Waste Generated	149	Metric Tons	-30% (from 214 in 2022)

The overall reduction in Scope 1 and 2 GHG emissions, totaling a 37% drop to 6,129 metric tons of $\text{CO}_2$e in 2023, is a strong positive trend. But, the massive increase in hazardous waste to 135 metric tons in 2023 is a clear near-term risk that demands focused action and investment in specialized waste management, especially as manufacturing scales up in 2025.

Focus on 'green lab initiatives' and recycling programs aims to mitigate the environmental footprint of research and production facilities.

To directly address the high-impact areas of R&D and manufacturing, Sarepta has implemented 'green lab initiatives' and recycling programs. These are essential for mitigating the high energy and waste intensity of laboratory work, where constant ventilation, cooling, and specialized equipment are necessary. You have to be defintely aggressive on lab efficiency to move the needle in biotech.

Concrete actions taken to reduce the environmental footprint of facilities include:

• Implementing nighttime and weekend setback programs for lighting and heating at most sites, including all laboratories.
• Undertaking a companywide re-lamping initiative to replace older compact fluorescent and metal halide bulbs with energy-efficient Light Emitting Diode (LED) technology in offices and the Andover, MA, site parking lot.
• Maintaining recycling programs for non-hazardous materials across facilities.

Environmental stewardship is a stated commitment in their corporate responsibility reporting.

Environmental stewardship is a stated core pillar in Sarepta Therapeutics' corporate responsibility reporting, a clear signal to investors and regulators that ESG (Environmental, Social, and Governance) factors are being formally integrated into their strategy. They recognize the vital connections between planetary health and human health, which is a strong narrative for a company focused on life-altering medicines. This commitment is supported by the formal tracking and measurement tools they've established to collect data on energy consumption, water use, material and hazardous waste, and GHG emissions, using 2022 as a baseline year. They are also evaluating their performance based on guidance from the Task Force on Climate-related Financial Disclosures (TCFD).

- Environmental stewardship is a stated commitment in their corporate responsibility reporting.

The company's commitment to environmental stewardship is a strategic move to manage long-term risk and align with investor expectations. Sarepta's overall net impact ratio, a measure of holistic value creation, is reported at a positive 62.3%, but this positive impact is achieved while causing negative impacts in categories like GHG emissions, highlighting the ongoing tension between their mission and their operational footprint. The next step for the company is to set concrete, time-bound reduction targets for waste and emissions that go beyond the 2023 baseline, particularly in the hazardous waste category.