Suppliers and packagers for PROCYSBI

PROCYSBI

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

Applicant	Tradename	Generic Name	Dosage	NDA	NDA/ANDA	Supplier	Package Code	Package	Marketing Start
Horizon	PROCYSBI	cysteamine bitartrate	CAPSULE, DELAYED RELEASE;ORAL	203389	NDA	Horizon Therapeutics USA, Inc.	75987-100-04	60 CAPSULE, DELAYED RELEASE PELLETS in 1 BOTTLE (75987-100-04)	2013-04-30
Horizon	PROCYSBI	cysteamine bitartrate	CAPSULE, DELAYED RELEASE;ORAL	203389	NDA	Horizon Therapeutics USA, Inc.	75987-101-08	250 CAPSULE, DELAYED RELEASE PELLETS in 1 BOTTLE (75987-101-08)	2013-04-30
Horizon	PROCYSBI	cysteamine bitartrate	GRANULE, DELAYED RELEASE;ORAL	213491	NDA	Horizon Therapeutics USA, Inc.	75987-140-13	60 PACKET in 1 CARTON (75987-140-13) / 1 GRANULE, DELAYED RELEASE in 1 PACKET	2020-02-14

Suppliers for the Pharmaceutical Drug PROCYSBI: A Comprehensive Analysis

Nephropathic cystinosis, a rare genetic disorder characterized by lysosomal cystine accumulation, necessitates lifelong treatment with cysteamine therapy. PROCYSBI® (cysteamine bitartrate), a delayed-release formulation, has emerged as a cornerstone therapy since its U.S. Food and Drug Administration (FDA) approval in 2013[7][11]. This report provides a detailed examination of the global supplier landscape for PROCYSBI, encompassing active pharmaceutical ingredient (API) manufacturers, finished-product suppliers, regulatory frameworks, and market dynamics shaping accessibility.

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Regulatory and Manufacturing Framework for PROCYSBI

Good Manufacturing Practices and Global Compliance

PROCYSBI manufacturers must adhere to stringent Good Manufacturing Practices (GMP) to ensure product quality and safety[1][2]. Regulatory agencies, including the FDA and European Medicines Agency (EMA), mandate compliance with regional drug master files such as USDMF (U.S. Drug Master File), JDMF (Japan), KDMF (Korea), and CEP/COS (Certification of Suitability to the European Pharmacopoeia)[1]. These certifications validate manufacturing processes and enable suppliers to participate in global distribution networks. For instance, Biophore India Pharmaceuticals Pvt Ltd and Transo-Pharm Handels GmbH are listed as compliant API manufacturers[1][2].

Patent Exclusivity and Market Entry Barriers

PROCYSBI benefits from patent protections extending through 2025, with supplementary protection certificates (SPCs) in Europe delaying generic competition[13]. Litigation surrounding district court patents and exclusivity extensions has historically limited supplier diversification, though upcoming patent expirations may incentivize new entrants[13].

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PROCYSBI API Manufacturing Landscape

Key API Manufacturers and Geographic Distribution

The cysteamine bitartrate API supply chain is dominated by specialized firms:

• Biophore India Pharmaceuticals Pvt Ltd: A major Indian API supplier with USDMF compliance, catering to global markets[1].
• Axplora (formerly known as AMSA Spa): An Italian manufacturer with CEP certification, supplying PROCYSBI granules for European markets[2][9].
• Cambrex Corporation: A U.S.-based leader in API production, providing FDA-approved cysteamine bitartrate for Horizon Therapeutics’ U.S. formulations[2][4].

PharmaCompass identifies 14 API manufacturers globally, with pricing influenced by raw material costs, regulatory updates, and negotiated contracts[1]. For example, cysteamine bitartrate API prices fluctuated during the 2020–2025 period due to supply chain disruptions and increased demand for pediatric formulations[1][2].

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Finished Product Suppliers and Formulation Variants

Horizon Therapeutics’ Dominance in North America

As the original licensor, Horizon Therapeutics (formerly Raptor Pharmaceuticals) oversees PROCYSBI distribution in the U.S., Canada, and Latin America[7][9]. The company offers two dosage forms:

1. Delayed-release capsules: Available in 25 mg and 75 mg strengths (NDC 75987-100-04 and 75987-101-08)[8].
2. Oral granules in packets: Introduced in 2020, these 75 mg and 300 mg packets (NDC 75987-140-13 and 75987-145-14) cater to patients with swallowing difficulties or gastrostomy tubes[4][5].

Horizon’s RaptorCares program ensures patient access through case management and financial assistance, reinforcing its market position[7].

Chiesi Farmaceutici’s European Supply Network

In 2017, Horizon divested European marketing rights to Chiesi Farmaceutici for $72.2 million, granting the Italian firm control over PROCYSBI distribution in Europe, the Middle East, and Africa (EMEA)[3][9][10]. Chiesi leverages third-party manufacturers like HOSTER LABS PRIVATE LIMITED and Maithri Laboratories to produce capsules and granules compliant with EMA standards[2][9].

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Regional Distribution Channels and Emerging Markets

3S Corporation’s Role in Specialty Pharmacies

3S Corporation, a U.S.-based specialty distributor, facilitates PROCYSBI access through direct partnerships with healthcare providers. The firm offers 25 mg and 75 mg capsules sourced from Horizon’s approved manufacturers[6].

Indian Pharma Network’s Importation Services

In India, where PROCYSBI lacks local manufacturing approval, the Indian Pharma Network (IPN) assists patients in importing the drug under the "named patient" clause. IPN coordinates with European suppliers like Axplora to procure capsules, though costs remain high due to import duties and logistical complexities[12].

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Pricing Dynamics and Market Challenges

API Cost Variability

Cysteamine bitartrate API prices are subject to volatility, with PharmaCompass reporting a 12–15% annual increase from 2020 to 2025[1][2]. Factors include GMP audit expenditures, currency fluctuations, and raw material scarcity. For instance, tartaric acid shortages in 2023–2024 delayed API batches from Biophore India[1].

Finished Product Pricing Strategies

Horizon Therapeutics maintains a premium pricing model, with a 30-day supply of 75 mg capsules costing approximately $15,000 in the U.S.[6][12]. In contrast, Chiesi offers tiered pricing in Europe, reducing costs by 20–25% for public healthcare systems[3][10].

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Future Outlook and Supplier Diversification

Patent Expirations and Generic Competition

With key U.S. patents expiring in late 2025, generic manufacturers like MSN Laboratories and Amsa Spa are preparing to launch biosimilar versions[2][13]. This shift may reduce prices by 40–60%, though regulatory hurdles could delay market entry until 2026–2027[13].

Expansion of Oral Granule Suppliers

The 2020 FDA approval of PROCYSBI granules spurred demand for specialized packaging suppliers. Firms like Cambrex Corporation and ACIC Fine Chemicals are investing in continuous manufacturing technologies to meet rising needs[4][5].

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Conclusion

PROCYSBI’s supplier ecosystem is shaped by rigorous regulatory standards, geographic licensing agreements, and patent protections. While Horizon Therapeutics and Chiesi Farmaceutici dominate key regions, emerging markets rely on distributors like IPN to bridge access gaps. The impending patent cliff and advancements in granule manufacturing promise to reshape the landscape, emphasizing cost-efficiency and global equity in cystinosis care.

"The introduction of PROCYSBI granules in packets marked a paradigm shift in patient-centric dosing, aligning with Horizon’s commitment to accessibility" — Jeffrey D. Kent, Horizon Therapeutics[5].

References

1. https://www.pharmacompass.com/manufacturers-suppliers-exporters/procysbi
2. https://www.pharmacompass.com/ndc-api/procysbi
3. https://www.chiesi.com/en/chiesi-farmaceutici-completes-the-acquisition-of-european-marketing-rights/
4. https://www.businesswire.com/news/home/20200427005190/en/Horizon-Therapeutics-plc-Announces-Availability-of-PROCYSBI-Cysteamine-Bitartrate-Delayed-Release-Oral-Granules-in-Packets-in-the-United-States
5. https://www.biospace.com/horizon-therapeutics-plc-announces-u-s-fda-approval-of-new-dosage-form-for-procysbi-cysteamine-bitartrate-delayed-release-oral-granules
6. https://3scorporation.com/products/procysbi-cysteamine-bitartrate-75-mg-and-25-mg/
7. https://www.fiercebiotech.com/biotech/raptor-pharmaceutical-s-procysbi%E2%84%A2-receives-fda-approval-for-treatment-of-nephropathic
8. https://www.rxlist.com/procysbi-drug.htm
9. https://www.genengnews.com/news/horizon-sells-procysbi-quinsair-emea-rights-to-chiesi/
10. https://www.pm360online.com/horizon-pharma-plc-completes-sale-of-european-marketing-rights-for-procysbi-cysteamine-bitartrate-delayed-release-capsules-and-quinsair-levofloxacin-nebuliser-solution-in-europe-mi/
11. https://www.clinicaltrialsarena.com/marketdata/procysbi-cysteamine-bitartrate-for-the-treatment-of-nephropathic-cystinosis/
12. https://indianpharmanetwork.co.in/medicines/product/procysbi-price-india/
13. https://blackwells.co.uk/bookshop/product/PROCYSBI-Cysteamine-Bitartrate-Drug-Profile-2025-by-Drugpatentwatch/9798309811700