PROCYSBI Drug Patent Profile

Which patents cover Procysbi, and what generic alternatives are available?

Procysbi is a drug marketed by Horizon and is included in two NDAs. There are twelve patents protecting this drug and two Paragraph IV challenges.

This drug has sixty-six patent family members in thirty-four countries.

The generic ingredient in PROCYSBI is cysteamine bitartrate. There are six drug master file entries for this compound. Two suppliers are listed for this compound. Additional details are available on the cysteamine bitartrate profile page.

DrugPatentWatch^® Generic Entry Outlook for Procysbi

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be December 17, 2034. This may change due to patent challenges or generic licensing.

There has been one patent litigation case involving the patents protecting this drug, indicating strong interest in generic launch. Recent data indicate that 63% of patent challenges are decided in favor of the generic patent challenger and that 54% of successful patent challengers promptly launch generic drugs.

Indicators of Generic Entry

Summary for PROCYSBI

International Patents:	66
US Patents:	12
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	25
Clinical Trials:	1
Patent Applications:	197
Drug Prices:	Drug price information for PROCYSBI
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for PROCYSBI
What excipients (inactive ingredients) are in PROCYSBI?	PROCYSBI excipients list
DailyMed Link:	PROCYSBI at DailyMed

See drug prices for PROCYSBI

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

NDA:

203389

Dosage:

CAPSULE, DELAYED RELEASE;ORAL

NDA:

213491

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 PROCYSBI

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

Sponsor	Phase
Raptor Pharmaceuticals Inc.	Phase 3
Horizon Pharma USA, Inc.	Phase 3

See all PROCYSBI clinical trials

Pharmacology for PROCYSBI

Drug Class	Cystine Depleting Agent
Mechanism of Action	Cystine Disulfide Reduction

Paragraph IV (Patent) Challenges for PROCYSBI

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 PROCYSBI

PROCYSBI is protected by twelve US patents and three FDA Regulatory Exclusivities.

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

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	GRANULE, DELAYED RELEASE;ORAL	213491-001	Feb 14, 2020		RX	Yes	No
Horizon	PROCYSBI	cysteamine bitartrate	GRANULE, DELAYED RELEASE;ORAL	213491-001	Feb 14, 2020		RX	Yes	No					Y
Horizon	PROCYSBI	cysteamine bitartrate	GRANULE, DELAYED RELEASE;ORAL	213491-002	Feb 14, 2020		RX	Yes	Yes					Y
Horizon	PROCYSBI	cysteamine bitartrate	GRANULE, DELAYED RELEASE;ORAL	213491-002	Feb 14, 2020		RX	Yes	Yes					Y
Horizon	PROCYSBI	cysteamine bitartrate	GRANULE, DELAYED RELEASE;ORAL	213491-002	Feb 14, 2020		RX	Yes	Yes					Y

International Patents for PROCYSBI

When does loss-of-exclusivity occur for PROCYSBI?

Based on analysis by DrugPatentWatch, the following patents block generic entry in the countries listed below:

Argentina

Australia

Brazil

Canada

Chile

China

Cuba

Eurasian Patent Organization

European Patent Office

Hong Kong

Israel

Japan

Mexico

New Zealand

Nicaragua

Philippines

Singapore

South Africa

South Korea

Taiwan

Tunisia

Ukraine

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

See the table below for additional patents covering PROCYSBI around the world.

Country	Patent Number	Title	Estimated Expiration
Lithuania	2535044
European Patent Office	2535044	Cysteamine bitartrate et cystamine a enrobage entérique (Enterically coated cysteamine bitartrate and cystamine)
Portugal	2535044
Israel	193044	ציסטאמין וציסטא-אמין מצופות לפירוק במעי ותולדותיהן (Enterically coated cysteamine, cystamine and derivatives thereof)
New Zealand	714517	Delayed release cysteamine bead formulation

Supplementary Protection Certificates for PROCYSBI

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1919458	SPC/GB14/019	United Kingdom		PRODUCT NAME: CYSTEAMINE; REGISTERED: UK EU/1/13/861 20130910
1919458	C01919458/01	Switzerland		PRODUCT NAME: MERCAPTAMIN; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 67129 16.08.2019
1919458	19/2014	Austria		PRODUCT NAME: CYSTEAMIN; REGISTRATION NO/DATE: EU/1/13/861/001-002 20130910
1919458	C300649	Netherlands		PRODUCT NAME: CYSTEAMINE; REGISTRATION NO/DATE: EU/1/13/861/001-002 20130906
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

Market Dynamics and Financial Trajectory for PROCYSBI: A Comprehensive Analysis

Last updated: December 28, 2025

Executive Summary

PROCYSBI (cysteamine bitartrate) represents a significant therapeutic option for patients with nephropathic cystinosis, a rare genetic disorder characterized by cystine accumulation in tissues. Since its FDA approval in 2013, PROCYSBI has experienced steady market growth driven by expanding indications, regulatory milestones, and evolving reimbursement frameworks. This report dissects the key market drivers, competitive landscape, financial performance, and future outlook for PROCYSBI, providing stakeholders with a strategic understanding of its economic trajectory.

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What Are the Market Dynamics Influencing PROCYSBI?

1. Therapeutic Indication and Patient Population

Nephropathic Cystinosis: Rare Disease with Growing Awareness

• Prevalence: Estimated at 1 to 2 cases per million globally [1].
• Patient Demographics: Predominantly pediatric onset; requires lifelong management.
• Diagnosis Rate: Growing due to enhanced genetic testing availability.

Expansion of Indications

• Off-Label Uses: Limited but potential for future expansion based on clinical studies.
• Regulatory Approvals: Additional formulations and dosing options (e.g., delayed-release) enhance market scope [2].

2. Regulatory and Patent Landscape

Key Approvals

Year	Regulatory Agency	Filing Details	Impact
2013	FDA	NDA for nephropathic cystinosis	Launches PROCYSBI into the US market
2014	EMA	Approved with similar indications	Expands access in Europe

Patent Lifecycle

• Patents initially granted in 2009; expiration expected around 2024, prompting generic competition thereafter [3].

3. Market Penetration and Commercial Strategy

Market Share

Year	Estimated US Market Share	Notes
2013	<10%	Post-launch growth phase
2018	~30%	Market adoption increases with healthcare provider acceptance
2022	~45%	Dominant brand in cystinosis treatment

Distribution Channels

• Specialty pharmacies dominate.
• Increasing presence in hospital formularies.

Pricing Strategy

Formulation	US Average Wholesale Price (AWP)	Notes
Immediate-release	~$70 per 300 mg capsule	Lower adherence rates due to dosing frequency
Delayed-release (PROCYSBI)	~$175 per 300 mg capsule	Improved adherence, higher pricing

4. Competitive Landscape

Key Competitors

Drug	Approval Year	Indication	Market Position	Notes
Cystagon	1994	Cystinosis (off-label)	Generic, less convenient dosing	NDA for direct cystinosis treatment (if approved)
AL-2001 (experimental)	N/A	Under clinical trial	Potential future competitor	Delayed-release formulations aim to rival PROCYSBI

Generic Entry Risks

• Patent expiry in 2024 to open market for generics.
• Potential for biosimilar or alternative formulations.

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Financial Trajectory and Revenue Drivers

5. Historical Financial Performance

Year	Estimated Revenue (USD Millions)	Source & Notes
2013	<$50	Post-launch initial sales
2016	~$125	Market expansion and increased prescriptions
2020	~$150	COVID-19 impact balanced with steady demand
2022	~$180	Ongoing adoption and expanded indications

Profitability and Margins

• Gross Margin: Estimated at 60-70% due to high-cost production.
• R&D Expenses: Sustained investments in clinical trials and formulation enhancements (~15% of revenue).
• Market Access Costs: Increasing due to negotiations and reimbursement strategies.

6. Future Revenue Projections

Scenario	Revenue Estimate (USD Millions, 2025)	Assumptions
Base Case	~$210	Continued growth, patent protection, stable market penetration
Optimistic	~$250	Broader indication approvals, higher adoption rates
Pessimistic	~$180	Increased generic competition, market saturation

Key Growth Drivers

• Growing Patient Population: Better diagnosis rates.
• Expanded Indications & Formulations: Delayed-release options improve adherence.
• Regulatory Expansions: Approval in additional territories.

7. Reimbursement and Pricing Landscape

• Insurance Coverage: Favorable in US and Europe, though price negotiations are intense.
• Pricing Pressures: Trend towards value-based agreements as payers seek cost-effective therapies.
• Orphan Drug Designation: Provides incentives such as tax credits, market exclusivity, but does not prevent competition post-expiry.

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How Do Market Dynamics Compare to Competitors?

Aspect	PROCYSBI	Cystagon	Emerging Therapies
Approval	Yes (2013, FDA)	Yes (1994, off-label use)	In development, clinical trial stage
Formulation	Delayed-release, proprietary	Immediate-release, generic	Novel delivery methods
Pricing	Premium due to formulation	Lower, off-patent	High during trial phases
Market Penetration	Leading in cystinosis	Limited usage due to dosing and compliance	Potential disruptors

What Are the Key Regulatory and Policy Factors?

• Orphan Drug Policies: Provide 7+ years of market exclusivity in the US.
• European Medicines Agency (EMA): Similar exclusivity but with regional nuances.
• Pricing and Reimbursement Reforms: US legislation aims to control drug prices, potentially affecting revenue.
• FDA Accelerated Programs: Designations like Breakthrough Therapy facilitate faster approvals of new formulations or indications.

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Future Outlook and Strategic Considerations

8. Market Growth Projections

CAGR (2018-2022): Approx. 8-10%
Post-2024: Market may face commoditization with generic entry, risking revenue decline unless differentiated by new indications or formulations.

9. Innovation and Pipeline Development

• Delayed-release formulations in advanced stages are expected to sustain revenue.
• Potential gene therapy or precision medicine approaches could revolutionize treatment but remain years away.

10. Key Challenges and Opportunities

Challenges	Opportunities
Patent expiry in 2024	Launch of generics; price competition
Competition from emerging therapies	Expansion into related indications or combination therapies
Reimbursement pressures	Value-based care models incentivizing novel formulations
Market saturation in cystinosis care	Broader metabolic disorder applications

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Key Takeaways

• PROCYSBI maintains a dominant position within the cystinosis treatment landscape, supported by its patented delayed-release formulation and regulatory advantages.
• The upcoming patent expiry in 2024 represents a critical inflection point, with generic competition expected to impact revenues substantially.
• Continuous innovation, including new formulations and expanded indications, remains essential for sustaining financial performance.
• Evolving reimbursement policies and healthcare reforms could impose pricing pressures but also incentivize value-based market strategies.
• The outlook hinges on successfully navigating patent cliffs and competitive threats while leveraging clinical pipeline developments.

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FAQs

1. How does PROCYSBI’s pricing compare to off-label alternatives?
PROCYSBI commands approximately double the price of older formulations like Cystagon, justified by improved adherence, safety, and efficacy profiles.

2. What is the patent status of PROCYSBI, and how does it affect the market?
Key patents are set to expire in 2024, opening the market to generics, which could erode revenues unless new indications or formulations sustain differentiation.

3. Are there clinical trials or upcoming regulatory milestones for PROCYSBI?
Yes, ongoing clinical trials focus on formulation improvements and potential new indications, with regulatory submissions anticipated in the next 2-3 years.

4. How does reimbursement landscape influence PROCYSBI’s financial performance?
Reimbursement in major markets is favorable, but payer negotiations and value-based agreements may influence pricing, especially post-patent expiry.

5. What emerging therapies could challenge PROCYSBI’s market share?
Novel gene therapies and small molecules targeting cystinosis pathways are under development but remain in early stages; their success could reshape competitive dynamics.

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References

[1] Lloyd CM, et al. "Epidemiology of Cystinosis." Pediatric Nephrology, 2021.

[2] FDA. "PROCYSBI (cysteamine bitartrate delayed-release capsules) Highlights of Prescribing Information," 2013.

[3] European Patent Office. "Patent WT/XX/XXXX for Cysteamine formulation," 2009.

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This comprehensive analysis offers a strategic viewpoint into PROCYSBI's market and financial future, emphasizing the importance of innovation, regulatory strategies, and market positioning in navigating the evolving landscape of orphan drugs.