Suppliers and packagers for generic pharmaceutical drug: PATISIRAN SODIUM

PATISIRAN SODIUM

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

Applicant	Tradename	Generic Name	Dosage	NDA	NDA/ANDA	Supplier	Package Code	Package	Marketing Start
Alnylam Pharms Inc	ONPATTRO	patisiran sodium	SOLUTION;INTRAVENOUS	210922	NDA	Alnylam Pharmaceuticals, Inc.	71336-1000-1	1 VIAL, SINGLE-DOSE in 1 CARTON (71336-1000-1) / 5 mL in 1 VIAL, SINGLE-DOSE	2018-08-13

Suppliers for the Pharmaceutical Drug: Patisiran Sodium

Last updated: July 30, 2025

Introduction

Patisiran sodium, marketed under the brand name Onpattro, is a groundbreaking RNA interference (RNAi) therapeutic developed for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). Since its approval by the U.S. Food and Drug Administration (FDA) in 2018, it has established itself as a pivotal therapy addressing a rare, life-threatening neurodegenerative condition. This article offers a comprehensive overview of the key suppliers for patisiran sodium, highlighting manufacturing sources, supply chain partners, and strategic considerations critical for stakeholders involved in procurement, distribution, and regulatory compliance.

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Manufacturers of Patisiran Sodium

1. Alnylam Pharmaceuticals

Alnylam Pharmaceuticals, headquartered in Cambridge, Massachusetts, is the pioneering developer and sole manufacturer of the patented active pharmaceutical ingredient (API) for patisiran sodium. As a leader in RNAi therapeutics, Alnylam maintains proprietary control over its production processes, including synthesis, modification, and encapsulation of the siRNA molecules that constitute patisiran.

Manufacturing Facilities & Capabilities:
Alnylam operates state-of-the-art manufacturing facilities that adhere to current Good Manufacturing Practices (cGMP), ensuring high-quality API production. These facilities are certified by regulatory authorities such as the FDA, EMA, and other regional bodies. Alnylam’s integrated supply chain encompasses raw material procurement, API synthesis, formulation, and packaging.

Strategic Importance:
Having control over its manufacturing processes allows Alnylam to ensure the integrity, purity, and stability of patisiran sodium, which are critical given the molecule’s complexity. The company’s capacity expansion and strategic partnerships aim to meet global demand, considering the rarity of hATTR amyloidosis.

2. Contract Manufacturing Organizations (CMOs)

While Alnylam retains exclusive responsibility for API development, it relies significantly on Contract Manufacturing Organizations to scale production and support global distribution. These partnerships are essential to mitigate supply chain risks and address manufacturing bottlenecks.

Examples of CMOs Involved:

• BioNTech: Involved in RNA-based vaccine production, with expertise in nucleic acid manufacturing, potentially supporting the synthesis of RNA molecules.
• Boehringer Ingelheim: A major CMO with extensive experience in biologics and complex therapeutics, offering large-scale GMP production.

The reliance on CMOs, although undisclosed in detail due to confidentiality, underscores the importance of established, high-quality partners capable of adhering to strict regulatory and quality standards.

3. Raw Material Suppliers

The synthesis of patisiran’s siRNA entails high-purity phosphoramidite nucleoside building blocks, specialized enzymes, and lipids for nanoparticle formulation.

• Nucleoside Suppliers:
  Major suppliers include Carbosynth, TriLink Biotechnologies, and ChemGenes — companies known for supplying pharmacopeial-grade phosphoramidite reagents.

• Lipid Nanoparticle (LNP) Components:
  Phospholipids, cholesterol, and PEG-lipids are obtained from suppliers like Avanti Polar Lipids, Lipoid GmbH, and CordenPharma.

• Enzymes & Modifiers:
  Specialized enzymes and chemical modifiers are sourced from biotech firms such as New England Biolabs and Sigma-Aldrich.

Supply Chain Dynamics & Challenges

The global supply for nucleic acid ingredients and lipid components is highly competitive and susceptible to geopolitical influences, regulatory fluctuations, and raw material shortages. The COVID-19 pandemic underscored vulnerabilities in complex biologics supply chains, prompting companies like Alnylam to diversify sourcing and build strategic inventories.

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Distribution Partners & Logistics

Post-manufacture, patisiran sodium distribution involves multiple layers:

• Distribution Partners:
  Alnylam collaborates with global logistics firms such as UPS Supply Chain Solutions and FedEx for temperature-controlled (cold chain) transportation.

• Regional Distributors:
  In markets like Europe, Japan, and emerging economies, local specialty distributors partner with Alnylam or licensed wholesalers to ensure seamless supply.

• Regulatory Considerations:
  Distribution channels must comply with regional regulations (e.g., EMA, PMDA), necessitating close coordination.

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Key Considerations for Stakeholders

• Regulatory Compliance & Certification:
  Suppliers must adhere to cGMP standards, with rigorous validation and documentation for quality assurance.

• Supply Chain Resilience:
  Reliance on a limited number of raw material suppliers poses risks, emphasizing the need for sourcing diversification.

• Pricing & Contract Terms:
  Given the high complexity and R&D investment, procurement negotiations often involve licensing agreements, royalty payments, and long-term supply contracts.

• Intellectual Property & Licensing:
  Alnylam maintains patents over the siRNA sequences and delivery technologies, impacting licensing arrangements for manufacturing rights.

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Future Outlook & Strategic Implications

Alnylam’s ongoing efforts to expand manufacturing capacity, including potential replication of production facilities and strategic alliances, aim to stabilize supply and reduce dependency risks. The development of biosimilar or alternative formulations could alter the supplier landscape in the future. Moreover, emerging players in RNAi delivery technologies could enter the supply chain, enhancing capacity and competition.

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Key Takeaways

• Alnylam Pharmaceuticals is the sole manufacturer of the API for patisiran sodium, supported by high-quality CMOs and raw material suppliers.

• The complexity of the manufacturing process necessitates rigorous quality standards, with the supply chain vulnerable to geopolitical and supply disruptions.

• Strategic partnerships and diversified sourcing are vital for ensuring consistent global access to patisiran sodium.

• Stakeholders should monitor regulatory developments, supply chain innovations, and patent landscapes to optimize procurement strategies.

• Continuous capacity expansion efforts by Alnylam are critical to meet the global demand for this rare disease therapy.

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FAQs

1. Who are the primary suppliers of raw materials for patisiran sodium?
Raw materials include phosphoramidite nucleosides, lipids, and enzymes, supplied by companies like Carbosynth, TriLink Biotechnologies, Avanti Polar Lipids, and Sigma-Aldrich.

2. Does Alnylam outsource manufacturing for patisiran sodium?
Yes, Alnylam primarily manufactures the API internally but partners with CMOs for large-scale production and worldwide distribution support.

3. What logistical challenges exist for distributing patisiran sodium globally?
The drug requires strict cold chain logistics, making transportation sensitive and reliant on capable logistics providers to maintain proper temperature control.

4. Are there any alternative suppliers or biosimilar options for patisiran sodium?
Currently, Alnylam holds exclusivity, with no approved biosimilars. The development of generic versions is hindered by patent protections and complex manufacturing technology.

5. How does supply chain disruption impact availability and pricing of patisiran sodium?
Disruption can lead to shortages, increased costs, and delays, emphasizing the need for diversified sourcing, capacity expansion, and strategic inventory management.

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References

1. Alnylam Pharmaceuticals. Patisiran (Onpattro) FDA approval press release. 2018.
2. U.S. Food and Drug Administration. FDA approves first-of-its-kind RNA interference drug to treat a rare genetic disease. 2018.
3. European Medicines Agency. Summary of Product Characteristics for Onpattro. 2019.
4. Industry reports on RNAi therapeutic manufacturing and supply chain management.
5. Cited raw material suppliers' technical datasheets and websites.

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This comprehensive overview aims to inform business professionals about the pivotal suppliers supporting the production and distribution of patisiran sodium, enabling informed decision-making amid an evolving therapeutic landscape.