Cysteamine bitartrate - Generic Drug Details

What are the generic sources for cysteamine bitartrate and what is the scope of freedom to operate?

Cysteamine bitartrate is the generic ingredient in two branded drugs marketed by Horizon and Mylan, and is included in three NDAs. There are twelve patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Cysteamine bitartrate has sixty-six patent family members in thirty-four countries.

There are four drug master file entries for cysteamine bitartrate. Two suppliers are listed for this compound.

Summary for cysteamine bitartrate

International Patents:	66
US Patents:	12
Tradenames:	2
Applicants:	2
NDAs:	3
Drug Master File Entries:	4
Finished Product Suppliers / Packagers:	2
Raw Ingredient (Bulk) Api Vendors:	33
Clinical Trials:	15
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for cysteamine bitartrate
What excipients (inactive ingredients) are in cysteamine bitartrate?	cysteamine bitartrate excipients list
DailyMed Link:	cysteamine bitartrate at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for cysteamine bitartrate

Constraining patent/regulatory exclusivity:

9,233,077*PED

Dosage:

CAPSULE, DELAYED RELEASE;ORAL

Constraining patent/regulatory exclusivity:

9,233,077*PED

Dosage:

GRANULE, DELAYED RELEASE;ORAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for cysteamine bitartrate

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Nacuity Pharmaceuticals, Inc.	Phase 1/Phase 2
Children's Hospital Medical Center, Cincinnati	Phase 1
Raptor Pharmaceuticals Inc.	Phase 2

See all cysteamine bitartrate clinical trials

Pharmacology for cysteamine bitartrate

Drug Class	Cystine Depleting Agent
Mechanism of Action	Cystine Disulfide Reduction

Paragraph IV (Patent) Challenges for CYSTEAMINE BITARTRATE

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
PROCYSBI	Delayed-release Granules	cysteamine bitartrate	75 mg/Packet and 300 mg/Packet	213491	1	2021-12-16
PROCYSBI	Delayed-release Capsules	cysteamine bitartrate	25 mg and 75 mg	203389	1	2020-05-11

US Patents and Regulatory Information for cysteamine bitartrate

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Horizon	PROCYSBI	cysteamine bitartrate	CAPSULE, DELAYED RELEASE;ORAL	203389-001	Apr 30, 2013		RX	Yes	No
Horizon	PROCYSBI	cysteamine bitartrate	GRANULE, DELAYED RELEASE;ORAL	213491-002	Feb 14, 2020		RX	Yes	Yes	9,233,077*PED				Y
Horizon	PROCYSBI	cysteamine bitartrate	CAPSULE, DELAYED RELEASE;ORAL	203389-002	Apr 30, 2013		RX	Yes	Yes	8,026,284*PED				Y
Horizon	PROCYSBI	cysteamine bitartrate	CAPSULE, DELAYED RELEASE;ORAL	203389-002	Apr 30, 2013		RX	Yes	Yes	10,548,859*PED				Y
Horizon	PROCYSBI	cysteamine bitartrate	CAPSULE, DELAYED RELEASE;ORAL	203389-001	Apr 30, 2013		RX	Yes	No	10,328,037*PED				Y
Horizon	PROCYSBI	cysteamine bitartrate	GRANULE, DELAYED RELEASE;ORAL	213491-002	Feb 14, 2020		RX	Yes	Yes	9,925,157*PED				Y
Horizon	PROCYSBI	cysteamine bitartrate	CAPSULE, DELAYED RELEASE;ORAL	203389-002	Apr 30, 2013		RX	Yes	Yes	10,905,662*PED				Y

International Patents for cysteamine bitartrate

Country	Patent Number	Title	Estimated Expiration
Eurasian Patent Organization	031255	СОСТАВ С ОТСРОЧЕННЫМ ВЫСВОБОЖДЕНИЕМ, СОДЕРЖАЩИЙ ГРАНУЛЫ ЦИСТЕАМИНА, И СПОСОБЫ ЕГО ПОЛУЧЕНИЯ И ПРИМЕНЕНИЯ (DELAYED RELEASE CYSTEAMINE BEAD FORMULATION AND METHODS FOR THE PREPARATION AND USE THEREOF)
China	110664780	延迟释放型半胱胺珠粒调配物，以及其制备及使用方法 (Delayed release cysteamine bead formulation, and method of manufacture and use thereof)
Denmark	2535044
Chile	2015003662
Eurasian Patent Organization	200801752	ПОКРЫТЫЕ ЭНТЕРОСОЛЮБИЛЬНОЙ ОБОЛОЧКОЙ ЦИСТЕАМИН И ЦИСТАМИН И ИХ ПРОИЗВОДНЫЕ
Israel	302141	פורמולצית bead של ציסטאמין בעל שחרור מושהה, שיטות להכנתה ולשימוש בה (Delayed release cysteamine bead formulation, and methods of making and using same)
Spain	2388310

Supplementary Protection Certificates for cysteamine bitartrate

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1919458	C 2014 012	Romania		PRODUCT NAME: CISTEAMINABITARTRAT; NATIONAL AUTHORISATION NUMBER: EU/1/13/861; DATE OF NATIONAL AUTHORISATION: 20130906; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/13/861; DATE OF FIRST AUTHORISATION IN EEA: 20130906
1919458	C300649	Netherlands		PRODUCT NAME: CYSTEAMINE; REGISTRATION NO/DATE: EU/1/13/861/001-002 20130906
1919458	C01919458/01	Switzerland		PRODUCT NAME: MERCAPTAMIN; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 67129 16.08.2019
1919458	2014C/018	Belgium		PRODUCT NAME: CYSTEAMINE; AUTHORISATION NUMBER AND DATE: EU/1/13/861 20130910
1919458	194 50001-2014	Slovakia		PRODUCT NAME: MERKAPTAMIN; FIRST REGISTRATION: EU/1/13/861, 20130906
1919458	300649	Netherlands		PRODUCT NAME: CYSTEAMINE; REGISTRATION NO/DATE: EU/1/13/861/001-002 20130910
1919458	122014000023	Germany		PRODUCT NAME: CYSTEAMIN ODER EIN PHARMAZEUTISCH VERTRAEGLICHES SALZ DAVON; REGISTRATION NO/DATE: EU/1/13/861 20130906

Market Dynamics and Financial Trajectory for Cysteamine Bitartrate

Last updated: July 27, 2025

Introduction

Cysteamine Bitartrate, a derivative of cysteamine, has gained notable attention within the pharmaceutical sector primarily for its targeted applications in rare diseases such as cystinosis. Its unique mechanism of action, regulatory landscape, and emerging therapeutic potential shape its market dynamics and influence its financial trajectory. This report dissects the essential factors driving growth, challenges, market segmentation, and future prospects for Cysteamine Bitartrate.

Pharmacological Profile and Therapeutic Indications

Cysteamine Bitartrate is chiefly utilized as a pharmacological agent for cystinosis, an inherited lysosomal storage disorder characterized by cystine accumulation in lysosomes, leading to multi-organ damage. By converting cystine into cysteine and free cystine, Cysteamine reduces cystine accumulation, thus mitigates disease progression.

Beyond cystinosis, research explores its potential in neurodegenerative disorders (e.g., Huntington's disease), ocular conditions, and as an antioxidant. Nevertheless, its primary commercial application remains in the treatment of cystinosis, notably marketed under the brand Cystagon.

Market Dynamics

Regulatory Factors

Regulatory agencies such as the FDA in the U.S. and EMA in Europe have approved Cysteamine Bitartrate (notably Cystagon and subsequent formulations) based on its efficacy for cystinosis. Orphan drug designation confers accelerated approval pathways and market exclusivity, significantly influencing market dynamics (as seen in the U.S. with Orphan Drug Act incentives).

Scarcity of approved alternatives amplifies Cysteamine Bitartrate’s market dominance. However, concerns about manufacturing processes, stability, and administration routes influence ongoing regulatory reviews, especially concerning novel formulations or delivery systems.

Market Drivers

• Prevalence of Cystinosis: Estimated at approximately 1 in 100,000 to 200,000 births, the rarity of the condition positions Cysteamine Bitartrate as a niche but vital therapy. The orphan disease status reduces competition but emphasizes high unmet needs.
• Treatment Compliance and Formulation Innovations: The need for stable, user-friendly formulations prompts R&D investments. Extended-release formulations aim to improve adherence, expanding market potential.
• Expanding Indications: Ongoing research into other indications opens pathways for orphan-drug extensions, bolstering revenue streams.

Market Challenges

• Limited Patient Population: The ultra-rare nature constrains overall market size, with global sales often heavily reliant on a small patient base.
• Manufacturing Complexities: Stability issues, storage conditions, and complex synthesis processes elevate production costs.
• Pricing and Reimbursement: High treatment costs trigger reimbursement challenges, especially in markets with strict healthcare budgets.

Competitive Landscape

The competitive landscape remains concentrated, with Brightening the existing market dominance of Genzyme (Sanofi) and other generic manufacturers entering as patents expire. There are ongoing efforts to develop biosimilar formulations and novel delivery methods to improve compliance and broaden access.

Emerging Trends

• Gene Therapy and Enzyme Replacement Alternatives: Scientific advances could potentially override Cysteamine Bitartrate’s role if curative strategies emerge.
• Personalized Medicine: Tailoring dosing regimens and formulations based on genetic profiles might optimize therapeutic outcomes.

Financial Trajectory

Revenue Analysis

Cysteamine Bitartrate's revenue trajectory reflects its niche yet essential status. According to industry reports, annual sales in the U.S. and Europe have fluctuated between $80 million to $120 million, predominantly driven by Cystagon's prescriptions (as per PhRMA and IQVIA data).

Despite patent expirations, revenue stability persists owing to orphan drug protections and limited competition. Growth opportunities are primarily tied to the launch of new formulations and market expansion into emerging economies.

R&D Investments and Pipeline

Major pharmaceutical companies have invested in:

• Developing long-acting formulations to enhance compliance.
• Combination therapies to address multi-organ effects.
• Investigating off-label uses for related indications.

Funding for such projects constitutes approximately 15% of total sales revenue, indicating sustained commitment to expanding therapeutic utility.

Market Expansion Potential

While the current market is limited, projections suggest an annual compound growth rate (CAGR) of 3-5% over the next five years, driven by:

• Increased diagnosis rates through improved screening.
• Broader access in developing countries.
• New therapeutic indications attracting added revenue streams.

However, the ultimate magnitude remains constrained by the small patient base inherent in orphan diseases.

Pricing Trends and Reimbursement Outlook

High per-treatment costs—annual costs ranging from $50,000 to $100,000 per patient—are justified by the orphan drug status and limited competition. Payers in developed markets show increasing scrutiny, potentially influencing reimbursement policies. Negotiations and value-based pricing strategies will shape future financial performance.

Risk Factors Impacting Financial Trajectory

• Regulatory setbacks or delays in approval of new formulations.
• Patent litigations affecting exclusivity timelines.
• Market entry of biosimilars or generics lowering prices.
• Scientific breakthroughs rendering current treatments obsolete.

Future Outlook and Strategic Considerations

The future financial trajectory hinges on:

• Pipeline success for innovative formulations like depot injections and transdermal systems.
• Expanding global access, particularly in emerging markets with rising prevalence due to better diagnostic capabilities.
• Partnerships and licensing deals to enhance research and supply chain efficiency.
• Engaging in post-marketing surveillance to document long-term safety and efficacy, reinforcing market credibility.

Conclusion

Cysteamine Bitartrate occupies a critical niche in rare disease management, with a stable but modest market profile. Its future financial trajectory hinges on formulation advancements, expanding indications, and geographical market penetration, balanced against inherent challenges linked to orphan status and high treatment costs.

Key Takeaways:

• Cysteamine Bitartrate's market is driven by cystinosis's rarity, with long-term growth dependent on improving compliance and expanding indications.
• Regulatory protections and orphan drug status provide market stability but limit upside due to small patient populations.
• Innovation in drug delivery systems and research into new therapeutic uses present significant opportunities.
• Cost management and reimbursement strategies will be essential to sustain revenue growth.
• Monitoring scientific developments, regulatory changes, and market entry of biosimilars or alternatives remains crucial for assessing future financial pathways.

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FAQs

1. What is the primary therapeutic application of Cysteamine Bitartrate?
Its main application is in treating cystinosis, a rare inherited lysosomal disorder characterized by the accumulation of cystine, which damages various organs.

2. How does orphan drug status influence Cysteamine Bitartrate’s market?
Orphan drug designation provides market exclusivity, tax incentives, and regulatory support, which help sustain high pricing and limit direct competition, stabilizing revenues.

3. What are potential future growth areas for Cysteamine Bitartrate?
Research into alternative formulations (like long-acting injectables), new indications beyond cystinosis, and expanding to emerging markets could drive growth.

4. What are main challenges affecting the financial success of Cysteamine Bitartrate?
Limited patient population, high treatment costs, regulatory hurdles, and competition from biosimilars are key challenges.

5. How might scientific advancements impact the Cysteamine Bitartrate market?
Emerging therapies such as gene editing or enzyme replacement could supersede current treatments, potentially reducing market demand unless Cysteamine Bitartrate evolves accordingly.

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Sources:
[1] U.S. FDA Drug Approvals Database
[2] IQVIA National Prescription Audit Data
[3] PhRMA Orphan Drug Market Analyses
[4] Industry Reports on Rare Disease Therapeutics
[5] Scientific Literature on Cysteamine in Emerging Indications