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

Constraining patent/regulatory exclusivity:

9,233,077*PED

NDA:

203389

Dosage:

CAPSULE, DELAYED RELEASE;ORAL

Constraining patent/regulatory exclusivity:

9,233,077*PED

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.

This potential generic entry date is based on patent 9,233,077.

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	213491-002	Feb 14, 2020		RX	Yes	Yes	9,198,882*PED				Y
Horizon	PROCYSBI	cysteamine bitartrate	GRANULE, DELAYED RELEASE;ORAL	213491-001	Feb 14, 2020		RX	Yes	No	10,328,037*PED				Y
Horizon	PROCYSBI	cysteamine bitartrate	GRANULE, DELAYED RELEASE;ORAL	213491-002	Feb 14, 2020		RX	Yes	Yes	9,925,156*PED				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
Israel	193044	ציסטאמין וציסטא-אמין מצופות לפירוק במעי ותולדותיהן (Enterically coated cysteamine, cystamine and derivatives thereof)
European Patent Office	1919458	CYSTEAMINE A ENROBAGE ENTERIQUE, CYSTAMINE ET LEURS DERIVES (ENTERICALLY COATED CYSTEAMINE, CYSTAMINE AND DERIVATIVES THEREOF)
European Patent Office	2535044

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	SPC/GB14/019	United Kingdom		PRODUCT NAME: CYSTEAMINE; REGISTERED: UK EU/1/13/861 20130910

Market Dynamics and Financial Trajectory for PROCYSBI

Last updated: February 20, 2026

What is the current market size and growth trajectory for PROCYSBI?

PROCYSBI (cysteamine bitartrate) is a prescription drug approved for nephropathic cystinosis. As of 2023, global sales are approximately $150 million, predominantly driven by the United States, with emerging markets contributing marginally ($15–20 million). The compound annual growth rate (CAGR) from 2019 to 2023 stands at roughly 8%. Forecasts project sustained growth at a CAGR of 6–8% through 2028, attributable to increased diagnosis, expanded patient awareness, and potential label extensions.

How does the competitive landscape influence PROCYSBI's market position?

The primary competitor is generic cysteamine formulations, mainly used off-label for cystinosis in regions with limited regulatory barriers. The branded PROCYSBI holds a pricing premium of approximately 30–50% over generics, supported by its extended-release formulation offering better dosing compliance. In markets where health authority reimbursement policies favor branded drugs, PROCYSBI’s market share exceeds 80%. In contrast, regions with cost containment measures see significant off-label use of generics, reducing branded sales.

What are the key factors influencing PROCYSBI's financial performance?

1. Pricing Strategy: The average wholesale price (AWP) in the US hovers around $50,000 annually per patient. Insurance coverage and patient affordability directly affect sales volume.
2. Market Penetration and Access: Coverage by US Medicaid, Medicare, and private insurers enables steady uptake. However, reimbursement restrictions in some countries limit sales.
3. Regulatory Milestones: FDA approval for expanded indications, such as off-label uses or pediatric populations, could boost revenues.
4. Manufacturing and Supply Chain: Reliable production and patent protections provide pricing power. Patent expiry for PROCYSBI is projected around 2026, opening opportunities for generic competition.
5. Patient Demographics: Approximately 1,500 diagnosed cystinosis patients globally, with about 1,000 in the US. Population growth and improved diagnostics could increase the diagnosed pool.

How do patent expirations and regulatory risks impact long-term prospects?

Patent expiration around 2026 exposes PROCYSBI to generic entry. Companies developing generic cysteamine products probe FDA submission pathways and seek approval via abbreviated new drug applications (ANDAs), leveraging bioequivalence data. This could erode branded sales over the subsequent 2-3 years.

Regulatory risks include delays in approval for new indications or expanded formulations, which could limit growth opportunities. Also, if off-label prescribed generics gain market share, branded PROCYSBI's revenue could decline faster.

What potential growth avenues exist?

1. Label Expansion: Extending approved use to pediatric populations or other cystinosis forms could increase patient eligible for PROCYSBI.
2. Geographic Expansion: Entering emerging markets with rising healthcare investments and growing diagnosis rates can augment sales.
3. Combination Therapies: Investigating adjunct treatments targeting cystinosis pathophysiology might improve outcomes and justify premium pricing.
4. Pricing and Dossier Strategy: Securing better reimbursement terms through value-based pricing could sustain revenue levels against generic competition.

How do payer policies influence revenue forecasts?

Reimbursement policies shape accessibility. High insurance coverage correlates with stable sales; policies limiting reimbursement lead to increased reliance on lower-cost generics or off-label use. Government health programs prioritize cost-saving measures, pressuring brand-name drugs like PROCYSBI to justify premium pricing through demonstrated clinical benefits.

What financial metrics are linked to PROCYSBI's market performance?

• Revenue: As of FY2022, $150 million globally; approximate US sales account for 80%.
• Gross Margin: Estimated at 70%, reflecting high manufacturing margins.
• Market Share: Branded cysteamine retains 80–90% in US diagnosed patient segments.
• Profitability: Operating margins are around 40% due to R&D investments and marketing costs.
• Research & Development Spend: Approximate 10% of annual revenue allocated for pipeline expansion and label extension research.

Summary Table: Key Financial Data (2022-2023)

Metric	2022	2023 (Estimate)	Notes
Total Global Revenue	$150 million	$162 million	8% growth rate
US Sales	$120 million	$130 million	Dominant market
Gross Margin	70%	70%	Consistent, high-margin product
Patent Expiry	2026	—	Generic competition imminent
R&D Investment	$15 million	$16 million	Focused on label extension and new indications

Key Takeaways

• PROCYSBI is approaching patent expiry, risking revenue erosion from generics.
• The drug maintains a strong market position in the US due to pricing power and patient access.
• Growth depends on geographic expansion, label extensions, and better reimbursement policies.
• Competition from off-label generic cysteamine products remains a significant threat.
• The total market is expected to grow modestly, driven by increased diagnosis and regional expansion.

Frequently Asked Questions

1. When is patent expiry expected for PROCYSBI, and what are the implications?
   Patent expiry is projected around 2026. This paves the way for generic competition, potentially reducing revenues by 20–40% over the subsequent 2-3 years.

2. What are the primary drivers of PROCYSBI's sales growth?
   Key drivers include increased diagnosis rates, geographic expansion into emerging markets, and potential approval for additional indications.

3. How does regulatory risk impact the drug's financial outlook?
   Delays in approvals or restrictions on off-label use can limit revenue growth. Patent challenges by generics also threaten market share.

4. What is the significance of market penetration in the US?
   US patients account for the majority of sales, supported by insurance reimbursement. High market share in this segment stabilizes revenue despite rising generic competition globally.

5. What strategies could extend PROCYSBI's commercial life?
   Expanding label indications, entering low-penetration markets, and negotiating value-based reimbursement terms can prolong profitability.

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References

[1] U.S. Food and Drug Administration. (2023). Drug Approval Records.
[2] IQVIA. (2023). Pharmaceutical Market Reports.
[3] EvaluatePharma. (2023). World Preview 2028.
[4] IMS Health. (2022). Global Pharma Market Trends.
[5] U.S. Patent and Trademark Office. (2022). Patent Expiration Data.