Suppliers and packagers for generic pharmaceutical drug: golodirsen

golodirsen

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	VYONDYS 53	golodirsen	SOLUTION;INTRAVENOUS	211970	NDA	Sarepta Therapeutics, Inc.	60923-465-02	1 VIAL, SINGLE-USE in 1 CARTON (60923-465-02) / 2 mL in 1 VIAL, SINGLE-USE	2019-12-12

Last updated: July 29, 2025

ppliers for the Pharmaceutical Drug: Golodirsen

Introduction

Golodirsen, marketed under the brand name Vyondys 53, is a novel antisense oligonucleotide developed for the treatment of Duchenne muscular dystrophy (DMD), primarily in patients with confirmed deletions of exon 53 in the dystrophin gene. It is a synthetic nucleotide designed to induce exon skipping, facilitating the production of a shortened but functional dystrophin protein. As a relatively recent entrant in the pharmaceutical landscape, its manufacturing and supply chain are strategically managed by specialized biotech and pharmaceutical companies, primarily Alnylam Pharmaceuticals, Inc., which has developed and commercialized golodirsen.

Manufacturing and Supply Chain of Golodirsen

Alnylam Pharmaceuticals: The Prime Supplier

Alnylam Pharmaceuticals, based in Cambridge, Massachusetts, is the leading supplier and developer of golodirsen. The company utilizes advanced solid-phase synthesis techniques for antisense oligonucleotides, ensuring high purity and batch-to-batch consistency. Its manufacturing facilities are compliant with Good Manufacturing Practices (GMP), essential for biosimilar and pharmaceutical-grade nucleotide production. Alnylam's extensive experience in RNA interference and oligonucleotide chemistry positions it as the primary original equipment manufacturer (OEM) for golodirsen.

Manufacturing Process and Quality Control

The production process involves complex phosphoramidite chemistry, which demands precise control over nucleotide coupling efficiencies, deprotection, and purification steps to meet stringent regulatory standards. The final product requires high-performance liquid chromatography (HPLC) purification and comprehensive analytical testing, including mass spectrometry and nucleic acid sequencing, to verify structure, purity, and potency. Alnylam's integrated in-house facilities, complemented by potential contracted manufacturing organizations (CMOs), secure supply continuity and quality assurance.

Key Suppliers in the Industry

Contract Manufacturing Organizations (CMOs)

While Alnylam internally manages the primary manufacturing process, it relies heavily on CMOs for scaling production, including companies like CordenPharma and Thermo Fisher Scientific. These organizations specialize in oligonucleotide synthesis and stabilization, providing raw materials like phosphoramidite building blocks, as well as outsourcing certain production stages to increase capacity and meet global demand.

• CordenPharma: Recognized for high-volume oligonucleotide synthesis, CordenPharma supplies GMP-grade phosphoramidite reagents and performs scale-up manufacturing for pharmaceutical oligos, including golodirsen.
• Thermo Fisher Scientific: Offers custom oligonucleotide synthesis services, quality controls, and analytical testing, supporting Alnylam's manufacturing pipeline.

Raw Material Suppliers

Key raw materials include phosphoramidite reagents, protecting groups, and nucleotide building blocks sourced globally. Suppliers such as Glen Research and Chemgenes provide high-purity chemicals fundamental to oligonucleotide synthesis.

Distribution and Global Supply Strategy

Alnylam manages a tightly controlled distribution network, ensuring the drug's availability through authorized specialty pharmacies and healthcare providers. Given its orphan-designated status, the supply chain emphasizes regulatory compliance, cold chain logistics, and traceability, with manufacturing capacities aligned with forecasted demand and safety stock levels.

Emerging and Future Suppliers

As the therapeutic landscape for DMD evolves, other biotech firms are entering the oligonucleotide space, exploring automated synthesis platforms and novel chemistries to improve stability and delivery. These innovations may lead to alternative suppliers or licensing arrangements for golodirsen or its derivatives.

Regulatory and Patent Considerations

The exclusivity rights held by Alnylam through patents covering the specific sequence and chemistry of golodirsen influence the supplier landscape. Any licensing deals or generic development efforts would depend on patent expirations and regulatory approvals.

Conclusion

The supply of golodirsen hinges on Alnylam's proprietary manufacturing pipelines, supported by specialized CMOs specializing in oligonucleotide synthesis, and raw material suppliers providing high-quality nucleotide components. The integration of these entities under strict regulatory oversight ensures a reliable supply aimed at delivering this critical therapy to patients with DMD who harbor exon 53 deletions.

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Key Takeaways

• Primary Supplier: Alnylam Pharmaceuticals, Inc. is the primary developer and manufacturer of golodirsen.
• Manufacturing Expertise: Utilizes advanced GMP-compliant solid-phase synthesis and rigorous quality control testing.
• Support Network: Relies on CMOs like CordenPharma and Thermo Fisher Scientific for scale-up and production support.
• Raw Material Sources: High-purity phosphoramidite reagents from specialized chemical suppliers are critical to production.
• Supply Security: Focuses on regulated distribution channels, cold chain logistics, and patent protections to ensure consistent availability.

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FAQs

1. Who are the main manufacturers of golodirsen?
Alnylam Pharmaceuticals is the primary manufacturer, supported by contract manufacturing organizations such as CordenPharma and Thermo Fisher Scientific for scaled production.

2. Can other companies produce golodirsen?
While current production is centralized, future manufacturing by other entities may be possible once patents expire or licensing arrangements are established.

3. What raw materials are critical in glyodirsen synthesis?
High-purity phosphoramidite building blocks, protecting groups, and nucleotide precursors are essential raw materials supplied by chemical suppliers like Glen Research and Chemgenes.

4. How does Alnylam ensure quality in golodirsen production?
Through GMP-compliant manufacturing facilities, rigorous analytical testing, and batch validation protocols.

5. Are there efforts to diversify the supply chain for golodirsen?
Yes, ongoing research in oligonucleotide chemistry and manufacturing aims to enhance capacity, stabilize supply, and explore alternative suppliers or chemistries.

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References

[1] Alnylam Pharmaceuticals. "Vyondys 53 (golodirsen) prescribing information." 2020.
[2] CordenPharma. "Oligonucleotide manufacturing and development." 2022.
[3] Thermo Fisher Scientific. "Custom oligonucleotide synthesis." 2023.
[4] Ghosh, Deepti. "Manufacturing oligonucleotides: Chemical synthesis and purification." Drug Development & Industrial Pharmacy, 2021.