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-two 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:	62
US Patents:	12
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	13
Clinical Trials:	1
Patent Applications:	15
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
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.

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	CAPSULE, DELAYED RELEASE;ORAL	203389-002	Apr 30, 2013		RX	Yes	Yes
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	9,192,590*PED				Y
Horizon	PROCYSBI	cysteamine bitartrate	GRANULE, DELAYED RELEASE;ORAL	213491-002	Feb 14, 2020		RX	Yes	Yes	9,925,158*PED				Y
Horizon	PROCYSBI	cysteamine bitartrate	GRANULE, DELAYED RELEASE;ORAL	213491-002	Feb 14, 2020		RX	Yes	Yes	9,173,851*PED				Y
Horizon	PROCYSBI	cysteamine bitartrate	GRANULE, DELAYED RELEASE;ORAL	213491-002	Feb 14, 2020		RX	Yes	Yes	10,548,859*PED				Y
Horizon	PROCYSBI	cysteamine bitartrate	GRANULE, DELAYED RELEASE;ORAL	213491-002	Feb 14, 2020		RX	Yes	Yes

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
China	105492000	Delayed release cysteamine bead formulation
Cyprus	1113690
Cyprus	1122943
European Patent Office	1919458	CYSTEAMINE A ENROBAGE ENTERIQUE, CYSTAMINE ET LEURS DERIVES (ENTERICALLY COATED CYSTEAMINE, CYSTAMINE AND DERIVATIVES THEREOF)
New Zealand	714517	Delayed release cysteamine bead formulation
Portugal	2535044
World Intellectual Property Organization (WIPO)	2007089670

Supplementary Protection Certificates for PROCYSBI

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1919458	19/2014	Austria		PRODUCT NAME: CYSTEAMIN; REGISTRATION NO/DATE: EU/1/13/861/001-002 20130910
1919458	2014C/018	Belgium		PRODUCT NAME: CYSTEAMINE; AUTHORISATION NUMBER AND DATE: EU/1/13/861 20130910
1919458	300649	Netherlands		PRODUCT NAME: CYSTEAMINE; REGISTRATION NO/DATE: EU/1/13/861/001-002 20130910
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	C01919458/01	Switzerland		PRODUCT NAME: MERCAPTAMIN; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 67129 16.08.2019
1919458	CR 2014 00013	Denmark		PRODUCT NAME: CYSTEAMIN, HERUNDER MERCAPTAMINBITARTRAT; REG. NO/DATE: EU/1/13/861 20130906
1919458	SPC/GB14/019	United Kingdom		PRODUCT NAME: CYSTEAMINE; REGISTERED: UK EU/1/13/861 20130910

Market Dynamics and Financial Trajectory for the Drug: PROCYSBI

Introduction

PROCYSBI, a delayed-release formulation of cysteamine, is a crucial treatment for nephropathic cystinosis, a rare genetic disorder. This article delves into the market dynamics and financial trajectory of PROCYSBI, highlighting its pricing controversies, revenue growth, and market impact.

Background on PROCYSBI

PROCYSBI is developed by Horizon Pharma (previously Raptor Pharmaceutical Corp.) and was launched on September 7, 2017. It replaced CYSTAGON, an immediate-release formulation that required dosing every six hours, with a more convenient 12-hour dosing regimen[1].

Market Size and Growth

The global market for cystinosis treatments, primarily driven by PROCYSBI and CYSTAGON, has shown significant growth. In 2017, the total market size for cystinosis in seven major markets was approximately USD 130.46 million, with the United States accounting for the largest share at USD 86.52 million[4].

Revenue Growth

PROCYSBI has demonstrated robust revenue growth since its launch. For the full year 2015, global net revenue for PROCYSBI was $94.2 million, representing a 36% increase from $69.5 million in 2014. This growth was driven by increased market penetration in the U.S. and Europe, as well as named patient sales in other territories[2].

In the fourth quarter of 2015, PROCYSBI generated $24.7 million in revenue, a 43% increase from the same period in 2014. Raptor Pharmaceutical Corp. projected further growth, setting a 2016 global net revenue guidance of $115 to $125 million, with an anticipated growth rate of 25%-30% year-over-year[2].

Pricing Controversies

One of the most significant challenges for PROCYSBI has been its pricing. In September 2022, the Patented Medicine Prices Review Board (PMPRB) in Canada determined that the price of PROCYSBI was excessively high. The PMPRB ordered Horizon Therapeutics Canada to reduce the price from $0.4140/mg to no higher than $0.2202/mg and to pay approximately $22 million in excess revenues to the Canadian government[1][3].

Regulatory Impact

The PMPRB's decision highlights the regulatory scrutiny that high-priced drugs, especially those for rare diseases, face. This case underscores the importance of price regulation to ensure that drugs remain accessible while allowing pharmaceutical companies to recoup their investment in research and development.

Cost of Sales and Operating Expenses

The financial performance of PROCYSBI is also influenced by its cost of sales and operating expenses. In 2015, the cost of sales for PROCYSBI was $12.6 million, up from $9.4 million in 2014, due to higher direct costs and royalty expenses associated with increased sales[2].

Raptor Pharmaceutical Corp. reported a net loss of $65.2 million for the full year 2015, despite the revenue growth. This loss was partly due to significant research and development, selling, general, and administrative expenses[2].

Market Outlook

The market for cystinosis treatments is expected to continue growing, driven by the increasing prevalence of the disease and the introduction of new therapies. The forecast period from 2019 to 2028 is anticipated to see significant growth, with potential therapies like AVR-RD-04, ELX-02, and A0003 contributing to the market expansion[4].

Competitive Landscape

PROCYSBI and CYSTAGON dominate the current market, but emerging therapies could alter the competitive landscape. The use of off-label therapies, such as hormonal treatments and renal cell transplantation, also plays a role in managing cystinosis, adding complexity to the market dynamics[4].

Financial Guidance and Projections

Historical financial guidance for PROCYSBI has been optimistic, with projections indicating continued revenue growth. However, the pricing controversy and subsequent regulatory actions have introduced uncertainty into these projections. Companies must balance revenue goals with regulatory compliance to ensure long-term sustainability.

Impact on Patients and Healthcare Systems

The pricing of PROCYSBI has significant implications for patients and healthcare systems. High drug prices can limit access to essential treatments, particularly for rare diseases where patient populations are small but treatment costs are high. The PMPRB's decision to reduce the price of PROCYSBI aims to make the drug more accessible while ensuring that the company can continue to invest in research and development[1][3].

Conclusion

The market dynamics and financial trajectory of PROCYSBI are complex, influenced by factors such as regulatory scrutiny, revenue growth, and the competitive landscape. Here are the key takeaways:

• Revenue Growth: PROCYSBI has shown significant revenue growth since its launch, driven by market penetration and sales in various territories.
• Pricing Controversies: The drug has faced regulatory challenges due to its high pricing, leading to a reduction in price and payment of excess revenues.
• Market Outlook: The market for cystinosis treatments is expected to grow, with emerging therapies potentially altering the competitive landscape.
• Financial Performance: The cost of sales and operating expenses are crucial factors in the financial performance of PROCYSBI.
• Regulatory Impact: Regulatory actions can significantly impact the pricing and accessibility of rare disease treatments.

FAQs

Q: What is PROCYSBI used for? A: PROCYSBI is used to treat nephropathic cystinosis, a rare genetic disorder.

Q: Who developed PROCYSBI? A: PROCYSBI was developed by Horizon Pharma (previously Raptor Pharmaceutical Corp.).

Q: What was the outcome of the PMPRB's review of PROCYSBI's pricing? A: The PMPRB found the price of PROCYSBI to be excessive and ordered a price reduction and the payment of approximately $22 million in excess revenues.

Q: How has the revenue for PROCYSBI grown since its launch? A: PROCYSBI has shown significant revenue growth, with a 36% increase in global net revenue from 2014 to 2015.

Q: What are the potential future therapies that could impact the market for cystinosis treatments? A: Emerging therapies such as AVR-RD-04, ELX-02, and A0003 are expected to contribute to the market expansion.

Sources

1. Smart & Biggar, PMPRB finds ultra-rare drug PROCYSBI is excessively priced, October 26, 2022.
2. GlobeNewswire, Raptor Pharmaceutical Corp. Reports Fourth Quarter and Full Year 2015 Financial Results and Provides 2016 Corporate Update, February 25, 2016.
3. Canada.ca, 2022–23 Departmental Results Report, November 9, 2023.
4. GlobeNewswire, Global Cystinosis Market Study 2017-2019 & 2020-2028: Epidemiology, Treatment, Management, Unmet Needs, Marketed & Emerging Drugs, Market Outlook, KOL Insights, January 27, 2020.