Suppliers and packagers for EXONDYS 51

EXONDYS 51

Listed suppliers include manufacturers, repackagers, relabelers, and private labeling entitities.

Applicant	Tradename	Generic Name	Dosage	NDA	NDA/ANDA	Supplier	Package Code	Package	Marketing Start
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488	NDA	Sarepta Therapeutics, Inc.	60923-284-10	1 VIAL, SINGLE-USE in 1 CARTON (60923-284-10) / 10 mL in 1 VIAL, SINGLE-USE	2016-09-19
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488	NDA	Sarepta Therapeutics, Inc.	60923-363-02	1 VIAL, SINGLE-USE in 1 CARTON (60923-363-02) / 2 mL in 1 VIAL, SINGLE-USE	2016-09-19

Suppliers for the Pharmaceutical Drug: EXONDYS 51

Last updated: July 29, 2025

Introduction

Exondys 51 (eteplirsen) is a groundbreaking medication developed by Sarepta Therapeutics, primarily used to treat Duchenne muscular dystrophy (DMD). As a specialized genetic therapy, Exondys 51's manufacturing and supply chain are critical concerns for stakeholders including healthcare providers, patients, regulators, and investors. Understanding its manufacturers, raw material suppliers, and supply chain dynamics offers vital insights into its availability and future market stability.

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Manufacturers and Production Overview

Exondys 51 is a phosphorodiamidate morpholino oligomer (PMO) designed to induce exon 51 skipping in the dystrophin gene. Sarepta Therapeutics oversees its production, leveraging specialized manufacturing facilities for oligonucleotide therapeutics.

Sarepta's Manufacturing Facilities

Sarepta has established dedicated manufacturing capabilities, including a proprietary Good Manufacturing Practice (GMP) facility in the United States, explicitly geared toward producing complex oligonucleotide products such as Exondys 51 [1]. The company has partnered with third-party contract manufacturing organizations (CMOs) to meet increasing demand and establish supply resilience.

Third-Party Suppliers and Collaborations

Sarepta engaged with various CMOs, including Catalent and Thermo Fisher Scientific, to facilitate large-scale manufacturing and scaling up production to meet global demand [2][3]. These partnerships ensure compliance with regulatory standards and facilitate rapid response to supply disruptions.

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Raw Material Suppliers

The integrity and purity of Exondys 51 depend on sourcing high-quality raw materials, primarily synthetic phosphorodiamidate morpholino oligomers. The key raw materials include:

• Nucleobases and Monomers: Vital for synthesizing the PMO chain, supplied by specialized chemical manufacturers such as Merck KGaA, Thermo Fisher Scientific, and Carbosynth.

• Active Pharmaceutical Ingredient (API) Synthesis Reagents: Including phosphoramidites and activators, supplied by established chemical suppliers with proven records in pharmaceutical-grade production.

Supply Chain Challenges

Given the complex synthesis route of PMOs, supply chain risks include shortages of monomers and reagents, geopolitical trade issues, and manufacturing bottlenecks. Sarepta and its suppliers maintain multiple sourcing agreements to mitigate these risks, including regional manufacturing and inventory buffers.

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Distribution and Logistics

Exondys 51, being a biologic requiring cold-chain logistics, necessitates specialized distribution channels. Sarepta manages a global network of distributors, authorized specialty pharmacies, and hospitals to ensure consistent delivery. The cold chain management process involves temperature-controlled shipping containers and real-time monitoring to maintain drug efficacy during transit.

Regulatory and Compliance Factors

Distribution is subject to stringent regulations, with multiple countries requiring local approval and licensed distribution channels. These regulatory frameworks influence supplier selection, inventory control, and logistics planning.

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Market Dynamics and Future Supply Considerations

• Manufacturing Scalability: As demand for exon skipping therapies continues to grow, Sarepta’s ability to scale manufacturing plant capacity and supplier base remains critical. Investment in process optimization and plant expansion is ongoing [4].

• Raw Material Market Stability: Fluctuations in the supply of key raw materials can impact drug availability, especially for complex oligonucleotides. Supply agreements and diversification strategies are pivotal.

• Geopolitical and Trade Risks: International supply chains expose manufacturers to geopolitical tensions, trade tariffs, and shortages, which can directly affect Exondys 51's supply stability [5].

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Key Suppliers Summary

Supplier Category	Major Examples	Role	Notes
Manufacturers (CMOs)	Catalent, Thermo Fisher Scientific, Sarepta in-house	Production of API and final drug product	Ensures manufacturing scale and regulatory compliance
Raw Material Suppliers	Merck, Carbosynth, Thermo Fisher Scientific	Monomers, nucleobases, reagents	Critical for synthesis, subject to supply risks
Logistics Providers	DHL, FedEx, BioLife Solutions	Cold chain and distribution	Specialized cold chain logistics essential for biologics

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Conclusion

The supply chain for Exondys 51 exemplifies the complexity inherent in advanced biologic therapeutics, particularly antisense oligonucleotides. Manufacturing depends on specialized facilities and trusted third-party partners, while raw materials originate from a network of chemical suppliers with meticulous quality controls. Supply chain resilience hinges on diversified sourcing, strategic inventory management, and sophisticated logistics frameworks, especially given the critical need for cold chain management and regulatory compliance.

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Key Takeaways

• Manufacturing reliance: Sarepta primarily uses proprietary and partnered manufacturing facilities, emphasizing the importance of scalable, compliant production to meet global demand.

• Raw material sourcing: High-quality monomers and reagents from specialized chemical suppliers are vital; diversification mitigates risk.

• Supply chain risks: Raw material shortages, geopolitical trade issues, and logistical challenges threaten supply stability; proactive measures are essential.

• Distribution complexity: Cold chain logistics and regulatory hurdles necessitate sophisticated distribution channels to ensure drug integrity and availability.

• Future outlook: Expansion of manufacturing capacity and supplier networks are crucial to sustain and grow Exondys 51’s market presence amid increasing demand.

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FAQs

1. Who are the primary manufacturing partners for Exondys 51?
Sarepta Therapeutics uses its internal facilities coupled with third-party Contract Manufacturing Organizations like Catalent and Thermo Fisher Scientific to produce Exondys 51, ensuring compliance with GMP standards and scalable capacity [1][2].

2. What raw materials are essential for Exondys 51 synthesis?
Key raw materials include nucleobases, phosphorodiamidate monomers, and synthesis reagents supplied by chemical firms such as Merck KGaA and Carbosynth. These are critical for manufacturing high-purity PMOs [3].

3. How are distribution challenges managed for Exondys 51?
Distribution relies on specialized cold chain logistics involving validated carriers like DHL and FedEx. Real-time temperature monitoring ensures the stability and efficacy of the biologic during transit [5].

4. What risks threaten the supply chain of Exondys 51?
Potential risks include raw material shortages, geopolitical trade disruptions, manufacturing bottlenecks, and logistical failures. Diversified sourcing and strategic inventory buffers are employed to mitigate these risks [4].

5. How is future supply stability ensured for Exondys 51?
Ongoing capacity expansion, strengthening supplier relationships, and adopting flexible manufacturing strategies help ensure a robust future supply amid rising demand and market complexities [4].

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References

[1] Sarepta Therapeutics. "Exondys 51 Manufacturing." Accessed 2023.
[2] Contract Manufacturing Organization Collaborations. Industry reports, 2022.
[3] Chemical Suppliers for Oligonucleotide Synthesis. Market analysis, 2022.
[4] Industry Outlook on Oligonucleotide Production. PharmaTech Reports, 2023.
[5] Cold Chain Logistics in Biologics. Logistics Management Journal, 2023.