Suppliers and packagers for generic pharmaceutical drug: TOFERSEN

TOFERSEN

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

Applicant	Tradename	Generic Name	Dosage	NDA	NDA/ANDA	Supplier	Package Code	Package	Marketing Start
Biogen Ma	QALSODY	tofersen	SOLUTION;INTRATHECAL	215887	NDA	Biogen Inc.	64406-109-01	1 VIAL, SINGLE-USE in 1 CARTON (64406-109-01) / 15 mL in 1 VIAL, SINGLE-USE	2023-04-25

Suppliers for the Pharmaceutical Drug: Tofersen

Last updated: July 28, 2025

Introduction

Tofersen, developed by Biogen Inc., is an antisense oligonucleotide (ASO) drug designed to treat amyotrophic lateral sclerosis (ALS) associated with mutations in the superoxide dismutase 1 (SOD1) gene. As a novel therapeutic, tofersen has garnered significant attention within the pharmaceutical and biotech industries, particularly concerning its sourcing, manufacturing, and supply chain management. Given the complexity inherent in oligonucleotide synthesis and the high standards for pharmaceutical-grade products, understanding the key suppliers involved in tofersen's production is critical for stakeholders—ranging from healthcare providers to investors. This article provides a comprehensive overview of the primary suppliers contributing to tofersen’s formulation, synthesis, delivery, and distribution.

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1. Raw Material Suppliers for Antisense Oligonucleotides (ASOs)

Nucleic Acid Building Blocks

The production of tofersen hinges on high-purity phosphorothioate-modified nucleotides, which serve as the foundational building blocks for ASOs.

• Chemical Suppliers: Major suppliers such as Jerini (formerly), Biontech, and Thermo Fisher Scientific provide modified nucleotides with the requisite chemical modifications for stability and efficacy.

• Specialized Nucleotide Suppliers: Companies like TriLink Biotechnologies and Lyophilized Nucleic Acids specialize in producing GMP-grade phosphorothioate-modified nucleotides necessary for clinical-grade ASO synthesis.

Quality and Compliance

Raw materials must adhere to stringent quality standards, including good manufacturing practice (GMP) compliance, to meet regulatory approval processes set by agencies like the FDA and EMA.

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2. Oligonucleotide Synthesis and Manufacturing

Contract Manufacturing Organizations (CMOs)

Due to the specialized nature of oligonucleotide synthesis, pharmaceutical companies often outsource production to experienced CMOs.

• Alnylam Pharmaceuticals' Manufacturing Partners: While Alnylam focuses on RNAi therapies, similar companies like Eurogentec and Corden Pharma serve as OEMs for complex oligonucleotide synthesis, providing custom synthesis and GMP manufacturing services.

• Agilent Technologies and Merck KGaA: These giants offer oligonucleotide synthesis equipment and services, supporting scale-up from research to commercial manufacturing.

Key Capabilities

• Phosphorothioate backbone incorporation
• 2'-O-methyl or 2'-O-methoxyethyl modifications
• High-throughput, GMP-compliant synthesis
• Purification processes, including high-performance liquid chromatography (HPLC)

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3. Formulation and Delivery Systems

Lipid Nanoparticle (LNP) Suppliers

Although tofersen is administered via intrathecal injection, formulation stability and delivery efficiency rest on advanced carrier systems.

• CordenPharma (a division of Merck KGaA): Provides formulation support for oligonucleotides, including LNPs or other delivery vectors compatible with CNS delivery.

• Bristol-Myers Squibb (BMS) and Moderna: These companies have developed lipid nanoparticles for RNA-based therapies and may serve as partners or benchmarks for specialized oligonucleotide delivery systems.

Specialized Delivery Technologies

• Alnylam's Delivery Technologies: Although focused on RNAi, Alnylam Technologies' expertise in lipid nanoparticle formulations guide delivery systems for similar oligonucleotide-based drugs like tofersen.

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4. Analytical and Quality Control Testing

Contract Testing Organizations

Quality assurance requires rigorous testing, including purity, potency, and stability assessments.

• Intertek and Eurofins Scientific: Global leaders in analytical testing of biopharmaceuticals, including oligonucleotides.

• PVS (PharmaVigilance Services): Provides custom analytical assays to certify batch purity and identity.

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5. Packaging and Distribution

Packaging Material Suppliers

Ensuring stability and integrity in storage and delivery involves specialized packaging.

• Nitto Denko and West Pharmaceutical Services: Suppliers of flexible tubing, vials, and sealing systems compatible with sterile, cold-chain distribution.

Distribution Partnerships

• Biogen’s Logistics Network: As the primary marketer and distributor of tofersen, Biogen leverages established supply chain partners to ensure cold-chain logistics, especially for intrathecally administered drugs requiring refrigeration.

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6. Regulatory and Compliance Support

Regulatory Consultants and Certification Bodies

Compliance with global standards requires collaboration with agencies and consultants.

• PharmaLex and Parexel: Offer regulatory consulting to ensure supplier compliance with GMP and other international standards.

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Conclusion

The supply chain for tofersen encapsulates a complex network of high-precision raw material providers, specialized oligonucleotide manufacturers, formulation experts, analytical testing organizations, and logistics firms. Ensuring GMP compliance, material purity, and delivery integrity remains paramount throughout the process. As biotech innovations continue to evolve, strategic partnerships with these suppliers will be instrumental in scaling production, maintaining supply, and expanding access to生命-saving therapies.

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Key Takeaways

• High-Quality Raw Materials Are Central: Suppliers of GMP-grade nucleotides like Thermo Fisher Scientific and TriLink underpin tofersen’s synthesis.
• Specialized Manufacturing Is Critical: Reliance on experienced CMOs such as Eurogentec ensures consistent, scalable oligonucleotide production.
• Delivery Systems Are Evolving: Lipid nanoparticle suppliers, including CordenPharma and Moderna, play crucial roles in optimizing CNS delivery.
• Regulatory and Quality Assurance Are Non-Negotiable: Testing organizations like Eurofins safeguard product integrity.
• Supply Chain Resilience Supports Accessibility: Strong collaborations across suppliers and logistics networks are vital for uninterrupted drug availability.

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FAQs

1. Who are the primary raw material suppliers for tofersen's active ingredient?
Suppliers like Thermo Fisher Scientific and TriLink Biotechnologies provide GMP-grade phosphorothioate-modified nucleotides crucial for tofersen synthesis.

2. Are there specific CMOs specializing in oligonucleotide manufacturing for clinical and commercial batches?
Yes, companies such as Eurogentec and Corden Pharma are experienced CMOs that produce GMP-grade oligonucleotides for therapeutic applications.

3. How is the delivery of tofersen optimized given its CNS target?
While tofersen is administered via intrathecal injection, formulation partners working with lipid nanoparticles or other carriers support stability and potentially enhance CNS penetration.

4. What role do analytical testing organizations play in tofersen's supply chain?
They verify the purity, potency, and stability of raw materials and finished products, ensuring regulatory compliance and patient safety.

5. How does Biogen ensure the logistical integrity of tofersen distribution?
Biogen coordinates with logistics providers equipped for cold-chain transportation, adhering to strict storage and handling protocols to maintain product efficacy until administration.

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Sources:

1. [FDA Approval and Clinical Trial Data on Tofersen]
2. [Biogen’s Official Documentation and Press Releases]
3. [Industry Reports on Oligonucleotide Supply Chains]
4. [Regulatory Guidelines on GMP Manufacturing of Oligonucleotides]
5. [Technical Publications on Oligonucleotide Delivery Systems]