PROCYSBI Drug Patent Profile

When do Procysbi patents expire, and when can generic versions of Procysbi launch?

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
Horizon Pharma USA, Inc.	Phase 3
Raptor Pharmaceuticals 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

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
Mexico	375377	FORMULACIÓN DE PERLAS DE CISTEAMINA DE LIBERACIÓN RETARDADA. (DELAYED RELEASE CYSTEAMINE BEAD FORMULATION)
Japan	6468661
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)

Supplementary Protection Certificates for PROCYSBI

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

Market Dynamics and Financial Trajectory for PROCYSBI

Last updated: July 27, 2025

Introduction

PROCYSBI (cysteamine bitartrate) represents a pivotal therapeutic advancement in the management of nephropathic cystinosis, a rare genetic disorder characterized by abnormal lysosomal cystine accumulation, leading to multiple organ damage. Market dynamics for PROCYSBI are shaped by its unique therapeutic profile, regulatory pathways, competitive landscape, and the evolving needs of rare disease drug development. Its financial trajectory offers insight into niche pharmaceutical markets, demonstrating how specialized products can generate substantial revenue despite limited patient populations.

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Market Overview and Therapeutic Profile

Nephropathic cystinosis affects approximately 1 in 100,000 to 200,000 individuals globally, primarily impacting children. It manifests as kidney Fanconi syndrome, corneal crystal accumulation, and multisystem organ damage. Left untreated, it results in renal failure, requiring transplantation, and significant morbidity.

PROCYSBI was approved by the U.S. Food and Drug Administration (FDA) in 2013 as a delayed-release formulation of cysteamine, designed to improve compliance with cysteamine therapy by reducing dosing frequency from multiple daily doses to twice daily. This pharmacokinetic enhancement improves patient adherence due to its more tolerable side effect profile and convenient dosing schedule.

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Market Dynamics

1. Demand Drivers

• Limited Patient Pool: The rarity of cystinosis imposes a constrained market. The global prevalence of diagnosed cases is approximately 1,500–2,000 patients, limiting potential sales volume.

• Unmet Medical Need: Prior to PROCYSBI, immediate-release cysteamine was the only treatment, requiring frequent dosing and causing gastrointestinal side effects, leading to poor adherence. PROCYSBI’s improved profile has increased treatment acceptance and persistence.

• Regulatory Incentives: Orphan drug designation in various regions has facilitated accelerated approval, market exclusivity, and tax benefits, thus incentivizing investment.

2. Competitive Landscape

• Immediate-Release Cysteamine: Generic formulations still dominate due to the absence of patent exclusivity for custom formulations outside brand protection periods.

• Other Emerging Therapies: As of today, no direct competitive drugs have gained regulatory approval for nephropathic cystinosis, positioning PROCYSBI as the primary branded option.

• Potential Future Competition: Gene therapy trials aim at cystinosis management, though clinical availability remains distant, maintaining PROCYSBI’s market dominance in the near term.

3. Pricing and Reimbursement

• Pricing Strategy: PROCYSBI commands premium pricing due to its delayed-release design and rare disease status. In the U.S., list prices approximate $45,000–$55,000 annually per patient.

• Coverage and Reimbursement: Insurance coverage varies, with major payers generally providing access due to its orphan drug designation and clinical necessity.

• Cost-Effectiveness Consideration: The high cost remains justified by its ability to prevent costly complications, hospitalizations, and transplantation, aligning with healthcare value frameworks.

4. Market Penetration and Adoption

• Physician and Patient Awareness: Educational efforts foster early diagnosis and treatment initiation, essential in a rare disease context.

• Adherence and Persistence: Twice-daily dosing improves compliance, potentially increasing lifetime patient retention and cumulative sales.

5. Regulatory and Policy Factors

• Orphan Drug Designation: Approved in multiple regions (FDA, EMA), facilitating market exclusivity until 2021 (U.S.) and 2024 (EU), with potential for extensions.

• Post-Marketing Commitments: Ongoing studies to expand indications and demonstrate long-term safety bolster market confidence.

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Financial Trajectory

1. Revenue Trends

Since its launch, PROCYSBI has demonstrated steady revenue growth, driven primarily by new patient initiation and ongoing treatment continuity among existing patients. In 2021, sales in the U.S. exceeded $occasional_placeholder$, with international markets contributing proportionally less due to regulatory and reimbursement barriers. Sales are expected to grow at a compound annual growth rate (CAGR) of approximately 5-7%, factoring in increased diagnosis, uptake, and geographic expansion.

2. R&D and Lifecycle Management

Pharmaceutical companies invest heavily in research to extend indications (e.g., for other cystinosis-related manifestations) and to improve formulations. Such efforts can extend market exclusivity, buffer revenue streams, and open new markets.

3. Patent and Exclusivity Impact

Patent protection on formulation and methods provides a period of market exclusivity, critical for recouping development expenses. Generic alternatives post-patent expiry threaten revenue streams, but patent extensions and new product variants can prolong profitability.

4. Collaboration and Licensing Agreements

Partnerships with specialty pharmacies, health authorities, and patient advocacy groups enhance market reach, particularly in emerging markets. Licensing agreements facilitate broader distribution, influencing revenues positively.

5. Future Outlook

• Market Expansion: Increasing diagnosis rates and treatment adherence could lead to revenue growth in the next 3-5 years, especially with expansion into untreated markets.

• Pipeline Development: The initiation of gene therapy and other innovative approaches may reshape the financial landscape, either complementing or competing with PROCYSBI.

• Pricing Pressures: Emerging biosimilars or alternative formulations could pressure pricing, impacting margins.

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Market Challenges and Opportunities

Challenges

• Limited Patient Base: Constraints imposed by the ultra-rare nature of cystinosis restrict scalable revenue.

• Pricing and Reimbursement Pressures: Payers are increasingly scrutinizing high-cost orphan drugs, potentially limiting reimbursement or negotiation leverage.

• Generic Competition: Entry of generic immediate-release cysteamine remains a threat post-patent expiry.

Opportunities

• Formulation Improvements: Development of even more patient-friendly formulations can capture additional market share.

• Indication Expansion: Use in non-nephropathic cystinosis or related disorders enhances revenue scope.

• Global Expansion: Growing awareness and diagnosis in emerging markets hold growth potential, especially with local manufacturing and subsidy programs.

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Key Takeaways

• PROCYSBI enjoys a strong leadership position in nephropathic cystinosis treatment, driven by improved pharmacokinetics and patient adherence.

• Its niche status limits overall market size but sustains high per-patient revenue, especially amid regulatory exclusivity periods.

• The primary challenge lies in balancing high pricing with payer acceptance amidst a small, geographically dispersed patient population.

• With ongoing efforts in formulation innovation, indication expansion, and market penetration, PROCYSBI’s revenue trajectory remains positive but subject to expiration of exclusivity and competition from future therapies.

• Strategic partnerships, global awareness campaigns, and health policy advocacy are pivotal in maximizing long-term financial performance.

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FAQs

Q1: How does PROCYSBI differentiate itself from immediate-release cysteamine?
A: PROCYSBI provides a delayed-release, twice-daily formulation that enhances compliance, reduces gastrointestinal side effects, and maintains consistent cysteamine plasma levels, thus improving treatment outcomes.

Q2: What is the primary factor limiting PROCYSBI’s market size?
A: Its market is constrained by the rarity of nephropathic cystinosis, affecting approximately 1-2 in 100,000 individuals worldwide.

Q3: How do patent protections impact PROCYSBI’s revenue prospects?
A: Patent protections and orphan drug exclusivity periods enable premium pricing and market control, but revenue growth diminishes once patents expire and generics enter the market.

Q4: Are there upcoming therapies that could threaten PROCYSBI’s market share?
A: Current pipelines focus on gene therapy and novel treatments; however, none have yet been approved. These could potentially disrupt the market once they reach commercialization.

Q5: What strategies could extend PROCYSBI’s market longevity?
A: Formulation enhancements, expanding indications, global market expansion, and securing regulatory exclusivity through patent extensions are key strategies.

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References

[1] Orphanet. Cystinosis. Accessed January 2023.
[2] U.S. Food and Drug Administration. PROCYSBI approval summary. 2013.
[3] EvaluatePharma. 2022. Orphan drug market insights.
[4] Cystinosis Foundation. Treatment guidelines. 2021.