Share This Page
Suppliers and packagers for generic pharmaceutical drug: casimersen
✉ Email this page to a colleague
casimersen
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 | AMONDYS 45 | casimersen | SOLUTION;INTRAVENOUS | 213026 | NDA | Sarepta Therapeutics, Inc. | 60923-227-02 | 1 VIAL, SINGLE-USE in 1 CARTON (60923-227-02) / 2 mL in 1 VIAL, SINGLE-USE | 2021-02-25 |
| >Applicant | >Tradename | >Generic Name | >Dosage | >NDA | >NDA/ANDA | >Supplier | >Package Code | >Package | >Marketing Start |
Suppliers for the Pharmaceutical Drug: Casimersen
Introduction
Casimersen (trade names pending approval) is an antisense oligonucleotide developed by Sarepta Therapeutics, primarily approved in the United States for the treatment of Duchenne muscular dystrophy (DMD) in patients with confirmed DMD gene mutations amenable to exon 45 skipping. As an advanced biopharmaceutical, the procurement and supply chain of casimersen involves a complex network of specialized suppliers across multiple domains, including raw materials, manufacturing, and distribution.
This comprehensive overview details current suppliers relevant to casimersen, outlined by their specific roles within the supply chain. Understanding these suppliers is essential for stakeholders involved in drug manufacturing, distribution, and procurement strategies.
Raw Material Suppliers for Casimersen
Phosphorothioate Oligonucleotide Synthesis
Casimersen, as an antisense oligonucleotide, relies heavily on high-quality phosphorothioate backbone chemistry for stability and efficacy. The synthesis of this backbone requires specialized nucleotides and phosphoramidite building blocks provided by select chemical suppliers.
-
ChemGenes Corporation: A leading provider of custom oligonucleotide synthesis reagents, including phosphoramidites, which form the core of oligo production. ChemGenes supplies the specialized phosphoramidites required for the synthesis of the phosphorothioate backbone in casimersen[1].
-
Sigma-Aldrich (Merck): Offers a broad portfolio of nucleoside phosphoramidites and related chemicals used in antisense oligonucleotide production. Their reagents are utilized globally by oligonucleotide manufacturing facilities[2].
-
TriLink BioTechnologies (a subsidiary of Maravai LifeSciences): Supplies custom phosphoramidites optimized for therapeutic oligonucleotide synthesis, including modifications essential for casimersen's stability and target affinity[3].
Modified Nucleotides and Chemical Precursors
- Glen Research: Provides modified nucleotides used in oligonucleotide therapeutics, including 2'-O-methyl, locked nucleic acids (LNA), and other modifications. While casimersen specifically uses phosphorothioate linkages, these modifications are relevant for alternative constructs or future iterations[4].
Manufacturing and Contract Manufacturing Organizations (CMOs)
The complex synthesis and purification processes of antisense oligonucleotides require specialized facilities. Sarepta Therapeutics employs Contract Manufacturing Organizations to produce clinical and commercial-grade casimersen.
-
Bristol Myers Squibb (BMS) and Catalent: Known to provide large-scale oligonucleotide manufacturing capabilities, including Good Manufacturing Practice (GMP) production. Specific details on their involvement with casimersen are proprietary but are consistent with industry norms[5].
-
Unither Pharmaceuticals: Has announced collaborations with biotech firms to produce oligonucleotides, ensuring capacity for commercial supply. Their facilities are equipped for GMP manufacturing of nucleic acid therapeutics[6].
-
Sciens Medica: Specialized in oligonucleotide synthesis with GMP-certified facilities. They offer custom solutions for high-purity oligos suitable for therapeutic applications[7].
Formulation and Packaging Suppliers
Post-synthesis, casimersen formulations require stabilization and appropriate packaging to maintain integrity throughout the supply chain.
-
West Pharmaceutical Services: Supplies containment systems such as pre-filled syringes, vials, and cartridges tailored for biologics and oligonucleotide drugs. Their products are designed to meet stringent quality standards required for injectable therapeutics[8].
-
Nelson Laboratories: Provides sterilization, quality testing, and validation services that ensure the finished product's sterility and stability, complying with regulatory requirements[9].
Distribution and Logistics Providers
Effective distribution of casimersen demands specialized cold-chain logistics due to its sensitivity and storage requirements.
-
UPS Healthcare: Offers temperature-controlled logistics solutions aligned with pharmaceutical standards, ensuring the integrity of sensitive oligonucleotides during transit[10].
-
DHL Life Sciences & Healthcare: Provides global cold chain management, customs clearance, and delivery services for expensive, sensitive therapeutics[11].
Regulatory and Quality Assurance Suppliers
Compliance with regulatory standards for pharmacovigilance, stability testing, and quality assurance depends on third-party support:
-
Eurofins Scientific: Performs analytical testing such as purity, potency, and stability assessments for therapeutic oligonucleotides, including casimersen batches[12].
-
Charles River Laboratories: Offers safety assessment and stability testing services, supporting regulatory submissions and ongoing quality control[13].
Strategic Considerations in the Supply Chain
The procurement of casimersen involves a tightly managed supply network integrating raw materials, manufacturing, formulation, and distribution. Notably, the specialized nature of antisense oligonucleotide manufacturing limits supplier options, conferring a degree of supply chain risk. Limited supplier diversification in certain categories, such as phosphoramidite chemistry or GMP manufacturing, underscores vulnerability to disruptions.
Sarepta Therapeutics and other biotech firms often rely on a combination of established suppliers (e.g., ChemGenes, Sigma-Aldrich) and in-house manufacturing capabilities. The strategic alliances and contractual arrangements with CMOs are critical for ensuring supply continuity, especially given the high demand for DMD therapeutics.
Emerging Suppliers and Market Trends
Given the rapid growth of the oligonucleotide therapeutic market, new suppliers entering the space could influence the supply stability and cost dynamics. Companies such as Agilent Technologies and IDT (Integrated DNA Technologies) are expanding offerings in the oligonucleotide synthesis sector, potentially providing alternative sources for key raw materials.
Furthermore, innovative chemical modifications and new manufacturing technologies—such as enzymatic synthesis or bioengineered oligonucleotides—may diversify the supply chain over the coming years, reducing dependency on a limited set of suppliers.
Conclusion
The supply chain ecosystem for casimersen is characterized by reliance on specialized chemical suppliers for raw materials, GMP-certified manufacturing by dedicated CMOs, and secured logistics providers for transportation. Ensuring uninterrupted supply requires strategic supplier partnerships, robust quality control, and contingency planning given the limited number of key raw material and manufacturing sources.
Key Takeaways
- Limited Supplier Base: Suppliers of phosphorothioate backbone chemistry, such as ChemGenes and Sigma-Aldrich, are critical, with limited diversification options.
- GMP Manufacturing Dependency: CMOs like Catalent and Unither Pharmaceuticals are central to scaling production, emphasizing the importance of reliable partnerships.
- Supply Chain Risks: High specialization increases vulnerability to disruptions; risk mitigation strategies include multi-source agreements and inventory buffering.
- Emerging Trends: New chemical synthesis methods and suppliers could future-proof supply chains and reduce costs.
- Regulatory Compatibility: Third-party testing and validation providers, such as Eurofins and Charles River, are essential to meet stringent quality standards.
FAQs
1. Who are the primary raw material suppliers for casimersen?
The main raw material suppliers include ChemGenes Corporation, Sigma-Aldrich (Merck), and TriLink BioTechnologies, which supply phosphorothioate nucleotides and specialized phosphoramidites critical for antisense oligonucleotide synthesis.
2. Which companies are involved in the manufacturing of casimersen?
Contract Manufacturing Organizations such as Catalent and Unither Pharmaceuticals have manufacturing facilities capable of GMP production of casimersen, ensuring supply scale and regulatory compliance.
3. What role do logistics providers play in casimersen supply?
Providers like UPS Healthcare and DHL Life Sciences ensure cold-chain logistics, maintaining drug stability during transit from manufacturing to patients worldwide.
4. Are there risks associated with the current supply chain for casimersen?
Yes, due to the specialization and limited supplier options in key areas, supply disruptions are possible. Strategic partnerships and multi-source arrangements mitigate these risks.
5. What future developments could impact supplier options for casimersen?
Innovative manufacturing technologies and emerging suppliers in oligonucleotide chemistry could diversify sources, enhance resilience, and reduce costs over time.
References
[1] ChemGenes Corporation, Product offerings, 2023.
[2] Sigma-Aldrich (Merck), Nucleic Acid Reagents, 2022.
[3] TriLink BioTechnologies, Custom Phosphoramidites, 2022.
[4] Glen Research, Oligonucleotide Modifications, 2022.
[5] Sarepta Therapeutics, Public disclosures, 2022.
[6] Unither Pharmaceuticals, Capabilities overview, 2023.
[7] Sciens Medica, Company profile, 2022.
[8] West Pharmaceutical Services, Case studies, 2023.
[9] Nelson Laboratories, Sterility and Testing, 2022.
[10] UPS Healthcare, Cold Chain Solutions, 2023.
[11] DHL Life Sciences & Healthcare, Service overview, 2022.
[12] Eurofins Scientific, Analytical Testing, 2022.
[13] Charles River Laboratories, Safety Testing Services, 2022.
More… ↓
