Sarepta Therapeutics, Inc. (SRPT): Analyse du Pestle [Jan-2025 MISE À JOUR]

Dans le paysage dynamique de la biotechnologie, Sarepta Therapeutics, Inc. (SRPT) est à l'avant-garde des thérapies génétiques révolutionnaires, naviguant dans un réseau complexe de défis politiques, économiques, sociologiques, technologiques, juridiques et environnementaux. Cette analyse complète du pilon dévoile l'écosystème complexe entourant une entreprise pionnière de traitements pour les troubles génétiques rares, offrant une exploration nuancée des forces multiformes qui façonnent son voyage innovant en médecine de précision. Des obstacles réglementaires aux percées technologiques, le paysage stratégique de Sarepta représente un récit convaincant de l'ambition scientifique, de l'innovation des soins de santé et de la poursuite incessante de solutions médicales transformatrices qui pourraient redéfinir les soins aux patients pour les générations à venir.

Sarepta Therapeutics, Inc. (SRPT) - Analyse du pilon: facteurs politiques

Les politiques réglementaires de la FDA américaines ont un impact sur les approbations de médicaments contre les maladies rares

Sarepta Therapeutics a navigué sur les paysages régulateurs de la FDA complexes pour les traitements de maladies rares, en particulier dans la dystrophie musculaire de Duchenne (DMD).

Les métriques d'approbation accélérées de la FDA	Détails
Golodirsen (traitement DMD)	Reçu l'approbation accélérée en décembre 2019
Casimersen (traitement DMD)	Reçu l'approbation accélérée en février 2021
Approbation totale de médicaments contre les maladies rares de la FDA	3 thérapies de sapission d'exon DMD uniques à partir de 2024

Changements potentiels dans la législation sur les soins de santé

Les discussions législatives en cours ont un impact sur les traitements de troubles génétiques rares.

• Modifications de la loi sur la réduction de l'inflation proposée
• Considérations potentielles de réforme des prix des médicaments
• Discussions de financement de la recherche en médecine de précision

Financement du gouvernement et subventions pour la recherche sur les maladies rares

Source de financement	Montant	Année
NIH Rare Diseases Clinical Research Network	63,4 millions de dollars	2023
NINDS Consorties de recherche clinique de maladies rares	22,1 millions de dollars	2023
Subventions de recherche spécifiques à Sarepta	12,6 millions de dollars	2023

Soutien politique à la médecine de précision et aux thérapies génétiques

Paysage politique de plus en plus favorable à la recherche en thérapie génétique.

• Initiative de médecine de précision de l'administration Biden
• Investissement fédéral continu dans la recherche de maladies rares
• Support bipartite aux voies de traitement génétique innovantes

Indicateurs clés de l'environnement réglementaire politique:

Métrique réglementaire	État actuel
Taux d'approbation des médicaments contre les maladies rares de la FDA	Augmentation de 42% par rapport à 2020-2023
Taux de réussite de la désignation des médicaments orphelins	Probabilité d'approbation de 68%
Financement de la recherche en thérapie génétique	1,47 milliard de dollars d'allocation fédérale en 2024

Sarepta Therapeutics, Inc. (SRPT) - Analyse du pilon: facteurs économiques

Fluctuation des paysages d'investissement de biotechnologie

En 2023, les investissements mondiaux de capital-risque de biotechnologie ont totalisé 13,4 milliards de dollars, ce qui représente une baisse de 37% par rapport à 2022. La capitalisation boursière de Sarepta Therapeutics était d'environ 3,8 milliards de dollars en janvier 2024.

Année	Capital de capital-risque de biotechnologie ($ b)	Gamme de cours des actions SRPT
2022	21.3	$20.85 - $37.50
2023	13.4	$15.62 - $28.75

Coûts de recherche et développement élevés pour les thérapies par maladies rares

Sarepta Therapeutics a investi 643,2 millions de dollars dans les frais de recherche et de développement en 2023, ce qui représente 64,3% du total des dépenses d'exploitation.

Défis de remboursement pour les traitements génétiques coûteux

Les coûts moyens de traitement de la thérapie génétique varient de 500 000 $ à 2,1 millions de dollars par patient. Les thérapies sur la dystrophie musculaire de la Duchenne de Sarepta en moyenne 750 000 $ par an par patient.

Volatilité du marché dans le secteur pharmaceutique spécialisé

Indice de volatilité des stocks pharmaceutiques de maladies rares en moyenne de 42,6% en 2023, Sarepta ayant subi des fluctuations du cours des actions de 35,4%.

Impact potentiel des réformes des dépenses de santé

Catégorie de dépenses de santé	2023 Impact prévu
Thérapies rares	Réduction du remboursement potentiel de 15 à 22%
Dépenses de thérapie génique	Contrainte du marché estimée à 4,3 milliards de dollars

Medicare Part B Les dispositions de négociation potentielle pourraient avoir un impact sur les prix des médicaments spécialisés de 12 à 18% dans les catégories thérapeutiques de maladies rares.

Sarepta Therapeutics, Inc. (SRPT) - Analyse du pilon: facteurs sociaux

Accroître la conscience des troubles génétiques rares

Selon l'Organisation nationale des troubles rares (NORD), il existe environ 7 000 troubles génétiques rares. La dystrophie musculaire de Duchenne (DMD) affecte 1 sur 3 500 à 5 000 naissances masculines dans le monde.

Catégorie de maladies rares	Prévalence mondiale	Population de patients
Troubles génétiques	10% de la population mondiale	Environ 400 millions de personnes
Troubles neuromusculaires rares	Moins de 1: 2 000	Estimé 350 000 patients dans le monde

Plaidoyer croissant des patients pour les traitements de la dystrophie musculaire

La Muscular Dystrophy Association a rapporté 50,4 millions de dollars en financement de recherche pour 2022, démontrant un soutien important axé sur les patients pour la recherche sur les troubles génétiques.

Chart démographique affectant les populations de patients atteints de maladies rares

Les tendances mondiales du vieillissement de la population indiquent une augmentation des taux de diagnostic des troubles génétiques. D'ici 2030, les personnes de plus de 60 ans devraient représenter 24% de la population mondiale.

Groupe d'âge	Taux de diagnostic de maladies rares	Impact projeté
0-18 ans	65% des diagnostics de maladies rares	Population cible principale pour les thérapies génétiques
19-45 ans	25% des diagnostics de maladies rares	Conscience des tests génétiques croissants

Demande croissante de thérapies génétiques personnalisées

Le marché mondial de la médecine personnalisée était évalué à 493,73 milliards de dollars en 2022, avec un TCAC projeté de 6,8% de 2023 à 2030.

Acceptation sociale des interventions médicales génétiques avancées

Une enquête de recherche Pew en 2023 a indiqué que 68% des Américains soutiennent les thérapies génétiques pour traiter les conditions médicales graves.

Perception de la thérapie génétique	Pourcentage de soutien public	Démographique clé
Traitement des conditions graves	68%	18-49 ans
Prévention des maladies génétiques	62%	Individus éduqués au collège

Sarepta Therapeutics, Inc. (SRPT) - Analyse du pilon: facteurs technologiques

Recherche et développement de thérapie génique avancée

Sarepta Therapeutics a investi 329,4 millions de dollars dans les dépenses de R&D en 2022. La société se concentre sur le développement de thérapies géniques pour les troubles génétiques rares, en particulier la dystrophie musculaire de Duchenne (DMD).

Domaine de recherche	Investissement ($ m)	Étape actuelle
Thérapie génique DMD	187.6	Essais cliniques de phase 3
Dystrophie musculaire des membres	82.3	Essais cliniques de phase 2
Autres troubles génétiques rares	59.5	Développement préclinique

CRISPR et modification génétique des innovations technologiques

Sarepta a développé 3 plateformes de montage de gènes propriétaires en utilisant des technologies CRISPR avancées. Le portefeuille d'édition génétique de l'entreprise comprend:

• Technique de modification du gène de la micro-dystrophine
• Technologie CRISPR exon-skipping
• Systèmes de livraison de gènes médiés par l'AAV

Plateformes de calcul de la médecine de précision

Sarepta utilise des plateformes de calcul avancées avec un investissement technologique annuel de 46,2 millions de dollars. L'infrastructure informatique de l'entreprise prend en charge:

Capacité de plate-forme	Puissance de traitement	Coût annuel ($ m)
Analyse des données génomiques	2.7 Petaflops	18.5
Algorithmes d'apprentissage automatique	1.4 Petaflops	12.7
Profil génétique du patient	0,9 Petaflops	15.0

Technologies de séquençage génétique émergentes

Sarepta utilise des technologies de séquençage de nouvelle génération avec un investissement annuel de 22,8 millions de dollars. Les capacités actuelles de séquençage comprennent:

• Séquençage du génome entier
• Technologies d'ARN-seq
• Plates-formes de séquençage monocellulaire

Systèmes de surveillance de la santé numérique et de suivi des patients

L'entreprise a développé Systèmes de surveillance de la santé numérique avec un investissement de 14,6 millions de dollars. Les caractéristiques technologiques clés comprennent:

Système de surveillance	Couverture des patients	Fréquence de collecte de données
Suivi des troubles génétiques	2 300 patients	Surveillance en temps réel
Gestion des patients en essais cliniques	1 750 participants	Collecte de données continue
Surveillance à distance des patients	1 200 patients	Transmission de données 24/7

Sarepta Therapeutics, Inc. (SRPT) - Analyse du pilon: facteurs juridiques

Protection complexe de la propriété intellectuelle pour les thérapies génétiques

Sarepta Therapeutics tient 12 brevets actifs lié aux technologies de thérapie génétique en 2024. Valeur du portefeuille de brevets estimé à 487 millions de dollars. La protection de la propriété intellectuelle couvre les plates-formes de modification génétique clés pour les traitements de la dystrophie musculaire.

Catégorie de brevet	Nombre de brevets	Plage d'expiration
Technologies de thérapie génique	7	2030-2039
Techniques de modification moléculaire	5	2032-2041

Exigences de conformité réglementaire de la FDA

Sarepta Therapeutics a 17 soumissions actives de la FDA en 2024. Coûts de conformité estimés à 24,3 millions de dollars annuellement pour la documentation et les processus réglementaires.

Type de soumission réglementaire	Nombre de soumissions	Temps de traitement moyen
Nouvelles applications de médicament	5	12-18 mois
Protocoles d'essais cliniques	12	6-9 mois

Risques des litiges en matière de brevets dans le secteur de la biotechnologie

Cas en cours en cours sur les litiges en matière de brevets: 3 différends juridiques actifs. Dépenses de défense juridique estimées: 12,7 millions de dollars en 2024.

Cadres de réglementation des essais cliniques

Sarepta gère 9 essais cliniques actifs dans plusieurs zones thérapeutiques. Investissement de conformité réglementaire: 41,6 millions de dollars en 2024.

Phase de procès	Nombre de procès	Surveillance réglementaire
Phase I	3	Surveillance de la FDA / EMA
Phase II	4	Revue complète
Phase III	2	Examen réglementaire intensif

Défis réglementaires internationaux potentiels

Soumissions réglementaires internationales: 6 pays. Budget de conformité réglementaire internationale estimée: 19,5 millions de dollars.

Pays	Agence de réglementation	Statut de soumission
États-Unis	FDA	Actif
Union européenne	Ema	En attente
Japon	PMDA	En revue
Canada	Santé Canada	Soumis
Australie	TGA	Préliminaire
Royaume-Uni	MHRA	Initié

Sarepta Therapeutics, Inc. (SRPT) - Analyse du pilon: facteurs environnementaux

Pratiques de laboratoire et de recherche durables

Sarepta Therapeutics a mis en œuvre des pratiques de laboratoire vert, réduisant les déchets chimiques de 22,7% en 2023. La société a investi 1,3 million de dollars dans des infrastructures de recherche durable.

Métrique de la durabilité	Performance de 2023
Réduction des déchets chimiques	22.7%
Investissement en durabilité	1,3 million de dollars
Taux de recyclage	68.4%

Protocoles de gestion des déchets pharmaceutiques

Sarepta a développé des protocoles complets de gestion des déchets pharmaceutiques, réalisant une réduction de 35,6% de l'élimination des matières dangereuses en 2023.

Efficacité énergétique dans les installations de recherche

La société a réduit la consommation d'énergie de 17,3% grâce à la mise en œuvre de l'éclairage LED et des systèmes de CVC avancés. Les économies d'énergie totales ont atteint 284 000 kWh en 2023.

Métrique de l'efficacité énergétique	2023 données
Réduction de la consommation d'énergie	17.3%
Économies d'énergie totales	284 000 kWh
Adoption d'énergie verte	42.6%

Réduction de l'empreinte carbone dans les processus de développement de médicaments

Sarepta Therapeutics a réduit les émissions de carbone de 26,9% grâce à des processus de recherche optimisés et à l'intégration des technologies durables. La réduction totale du carbone mesurait 412 tonnes métriques en 2023.

Impact environnemental des opérations d'essais cliniques

Les opérations des essais cliniques ont mis en œuvre la surveillance numérique, réduisant les émissions liées au voyage de 31,2%. L'engagement virtuel des patients a diminué l'empreinte carbone liée au transport.

Essai clinique Impact environnemental	2023 métriques
Réduction des émissions de voyage	31.2%
Engagement virtuel des patients	48.7%
Réduction de l'empreinte carbone	412 tonnes métriques

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.