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Last Updated: December 22, 2024

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PROCYSBI Drug Patent Profile


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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

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Drug patent expirations by year for PROCYSBI
Drug Prices for PROCYSBI

See drug prices for PROCYSBI

DrugPatentWatch® Estimated Loss of Exclusivity (LOE) Date for PROCYSBI
Generic Entry Dates for PROCYSBI*:
Constraining patent/regulatory exclusivity:
NDA:
Dosage:
CAPSULE, DELAYED RELEASE;ORAL
Generic Entry Dates for PROCYSBI*:
Constraining patent/regulatory exclusivity:
NDA:
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

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

See all PROCYSBI clinical trials

Pharmacology for PROCYSBI
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.

Based on analysis by DrugPatentWatch, the earliest date for a generic version of PROCYSBI is ⤷  Subscribe.

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 213491-001 Feb 14, 2020 RX Yes No ⤷  Subscribe ⤷  Subscribe ⤷  Subscribe
Horizon PROCYSBI cysteamine bitartrate GRANULE, DELAYED RELEASE;ORAL 213491-001 Feb 14, 2020 RX Yes No 9,192,590*PED ⤷  Subscribe Y ⤷  Subscribe
Horizon PROCYSBI cysteamine bitartrate GRANULE, DELAYED RELEASE;ORAL 213491-002 Feb 14, 2020 RX Yes Yes 9,925,158*PED ⤷  Subscribe Y ⤷  Subscribe
Horizon PROCYSBI cysteamine bitartrate GRANULE, DELAYED RELEASE;ORAL 213491-002 Feb 14, 2020 RX Yes Yes 9,173,851*PED ⤷  Subscribe Y ⤷  Subscribe
Horizon PROCYSBI cysteamine bitartrate GRANULE, DELAYED RELEASE;ORAL 213491-002 Feb 14, 2020 RX Yes Yes 10,548,859*PED ⤷  Subscribe Y ⤷  Subscribe
Horizon PROCYSBI cysteamine bitartrate GRANULE, DELAYED RELEASE;ORAL 213491-002 Feb 14, 2020 RX Yes Yes ⤷  Subscribe ⤷  Subscribe ⤷  Subscribe
>Applicant >Tradename >Generic Name >Dosage >NDA >Approval Date >TE >Type >RLD >RS >Patent No. >Patent Expiration >Product >Substance >Delist Req. >Exclusivity Expiration

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

Patent: 6628
Patent: FORMULACIÓN EN PERLAS DE CISTEAMINA DE LIBERACIÓN RETARDADA Y MÉTODOS DE PREPARACIÓN Y USO DE ELLA
Estimated Expiration: ⤷  Subscribe

Australia

Patent: 14281702
Patent: Delayed release cysteamine bead formulation
Estimated Expiration: ⤷  Subscribe

Brazil

Patent: 2015031417
Patent: forma de dosagem farmacêutica, composição farmacêutica, métodos para tratamento de um paciente com necessidade de cisteamina e para preparo de uma forma de dosagem
Estimated Expiration: ⤷  Subscribe

Canada

Patent: 14770
Patent: PREPARATION DE BILLES DE CYSTEAMINE A LIBERATION RETARDEE (DELAYED RELEASE CYSTEAMINE BEAD FORMULATION, AND METHODS OF MAKING AND USING SAME)
Estimated Expiration: ⤷  Subscribe

Patent: 38644
Patent: PREPARATION DE BILLES DE CYSTEAMINE A LIBERATION RETARDEE (DELAYED RELEASE CYSTEAMINE BEAD FORMULATION, AND METHODS OF MAKING AND USING SAME)
Estimated Expiration: ⤷  Subscribe

Chile

Patent: 15003662
Patent: Formulación en perlas de cisteamina de liberación retardada.
Estimated Expiration: ⤷  Subscribe

China

Patent: 5492000
Patent: Delayed release cysteamine bead formulation
Estimated Expiration: ⤷  Subscribe

Patent: 0664780
Patent: 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)
Estimated Expiration: ⤷  Subscribe

Cuba

Patent: 150178
Patent: FORMULACIÓN EN PERLAS DE CISTEAMINA DE LIBERACIÓN RETARDADA
Estimated Expiration: ⤷  Subscribe

Eurasian Patent Organization

Patent: 1255
Patent: 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)
Estimated Expiration: ⤷  Subscribe

Patent: 1690036
Patent: СОСТАВ С ОТСРОЧЕННЫМ ВЫСВОБОЖДЕНИЕМ, СОДЕРЖАЩИЙ ГРАНУЛЫ ЦИСТЕАМИНА, И СПОСОБЫ ЕГО ПОЛУЧЕНИЯ И ПРИМЕНЕНИЯ
Estimated Expiration: ⤷  Subscribe

European Patent Office

Patent: 10491
Patent: PRÉPARATION DE BILLES DE CYSTÉAMINE À LIBÉRATION RETARDÉE (DELAYED RELEASE CYSTEAMINE BEAD FORMULATION)
Estimated Expiration: ⤷  Subscribe

Patent: 39574
Patent: PRÉPARATION DE BILLES DE CYSTÉAMINE À LIBÉRATION RETARDÉE (DELAYED RELEASE CYSTEAMINE BEAD FORMULATION)
Estimated Expiration: ⤷  Subscribe

Hong Kong

Patent: 18066
Patent: 延遲釋放型半胱胺珠粒調配物 (DELAYED RELEASE CYSTEAMINE BEAD FORMULATION)
Estimated Expiration: ⤷  Subscribe

Israel

Patent: 4823
Patent: פורמולצית bead של ציסטאמין בעל שחרור מושהה, שיטות להכנתה ולשימוש בה (Delayed release cysteamine bead formulation, and methods of making and using same)
Estimated Expiration: ⤷  Subscribe

Patent: 2141
Patent: פורמולצית bead של ציסטאמין בעל שחרור מושהה, שיטות להכנתה ולשימוש בה (Delayed release cysteamine bead formulation, and methods of making and using same)
Estimated Expiration: ⤷  Subscribe

Japan

Patent: 68661
Estimated Expiration: ⤷  Subscribe

Patent: 16523250
Patent: 遅延放出システアミンビーズ処方、ならびにその作製方法および使用方法
Estimated Expiration: ⤷  Subscribe

Mexico

Patent: 15017366
Patent: FORMULACION DE PERLAS DE CISTEAMINA DE LIBERACION RETARDADA. (DELAYED RELEASE CYSTEAMINE BEAD FORMULATION.)
Estimated Expiration: ⤷  Subscribe

New Zealand

Patent: 4517
Patent: Delayed release cysteamine bead formulation
Estimated Expiration: ⤷  Subscribe

Nicaragua

Patent: 1500177
Patent: FORMULACIÓN EN PERLAS DE CISTEAMINA DE LIBERACIÓN RETARDADA
Estimated Expiration: ⤷  Subscribe

Philippines

Patent: 015502783
Patent: DELAYED RELEASE CYSTEAMINE BEAD FORMULATION
Estimated Expiration: ⤷  Subscribe

Singapore

Patent: 201510126Q
Patent: DELAYED RELEASE CYSTEAMINE BEAD FORMULATION
Estimated Expiration: ⤷  Subscribe

South Africa

Patent: 1508783
Patent: DELAYED RELEASE CYSTEAMINE BEAD FORMULATION
Estimated Expiration: ⤷  Subscribe

South Korea

Patent: 2281747
Estimated Expiration: ⤷  Subscribe

Patent: 2466253
Estimated Expiration: ⤷  Subscribe

Patent: 160045053
Patent: 서방성 시스테아민 비드 투약 형태 (DELAYED RELEASE CYSTEAMINE BEAD FORMULATION)
Estimated Expiration: ⤷  Subscribe

Patent: 210094140
Patent: 서방성 시스테아민 비드 투약 형태 (DELAYED RELEASE CYSTEAMINE BEAD FORMULATION)
Estimated Expiration: ⤷  Subscribe

Taiwan

Patent: 49100
Estimated Expiration: ⤷  Subscribe

Patent: 1534357
Patent: Delayed release cysteamine bead formulation, and methods of making and using same
Estimated Expiration: ⤷  Subscribe

Tunisia

Patent: 15000549
Patent: DELAYED RELEASE CYSTEAMINE BEAD FORMULATION
Estimated Expiration: ⤷  Subscribe

Ukraine

Patent: 7833
Patent: СКЛАД З ВІДСТРОЧЕНИМ ВИВІЛЬНЕННЯМ, ЩО МІСТИТЬ ГРАНУЛИ ЦИСТЕАМІНУ, І СПОСОБИ ЙОГО ОДЕРЖАННЯ ТА ЗАСТОСУВАННЯ (DELAYED RELEASE CYSTEAMINE BEAD FORMULATION)
Estimated Expiration: ⤷  Subscribe

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
Ukraine 117833 СКЛАД З ВІДСТРОЧЕНИМ ВИВІЛЬНЕННЯМ, ЩО МІСТИТЬ ГРАНУЛИ ЦИСТЕАМІНУ, І СПОСОБИ ЙОГО ОДЕРЖАННЯ ТА ЗАСТОСУВАННЯ (DELAYED RELEASE CYSTEAMINE BEAD FORMULATION) ⤷  Subscribe
Eurasian Patent Organization 200801752 ПОКРЫТЫЕ ЭНТЕРОСОЛЮБИЛЬНОЙ ОБОЛОЧКОЙ ЦИСТЕАМИН И ЦИСТАМИН И ИХ ПРОИЗВОДНЫЕ ⤷  Subscribe
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) ⤷  Subscribe
Japan 2016523250 遅延放出システアミンビーズ処方、ならびにその作製方法および使用方法 ⤷  Subscribe
World Intellectual Property Organization (WIPO) 2014204881 ⤷  Subscribe
Eurasian Patent Organization 023971 ПРИМЕНЕНИЕ КОМПОЗИЦИИ, СОДЕРЖАЩЕЙ ПОКРЫТЫЕ ЭНТЕРОСОЛЮБИЛЬНОЙ ОБОЛОЧКОЙ ЦИСТАМИН ИЛИ ЦИСТЕАМИН, ДЛЯ ЛЕЧЕНИЯ ЦИСТИНОЗА И СПОСОБ ЛЕЧЕНИЯ ЦИСТИНОЗА (USE OF A COMPOSITION COMPRISING ENTERICALLY COATED CYSTAMINE OR CYSTEAMINE FOR TREATING CYSTINOSIS AND METHOD OF TREATING CYSTINOSIS) ⤷  Subscribe
>Country >Patent Number >Title >Estimated Expiration

Supplementary Protection Certificates for PROCYSBI

Patent Number Supplementary Protection Certificate SPC Country SPC Expiration SPC Description
1919458 19/2014 Austria ⤷  Subscribe PRODUCT NAME: CYSTEAMIN; REGISTRATION NO/DATE: EU/1/13/861/001-002 20130910
1919458 300649 Netherlands ⤷  Subscribe PRODUCT NAME: CYSTEAMINE; REGISTRATION NO/DATE: EU/1/13/861/001-002 20130910
1919458 C300649 Netherlands ⤷  Subscribe PRODUCT NAME: CYSTEAMINE; REGISTRATION NO/DATE: EU/1/13/861/001-002 20130906
1919458 2014C/018 Belgium ⤷  Subscribe PRODUCT NAME: CYSTEAMINE; AUTHORISATION NUMBER AND DATE: EU/1/13/861 20130910
1919458 SPC/GB14/019 United Kingdom ⤷  Subscribe PRODUCT NAME: CYSTEAMINE; REGISTERED: UK EU/1/13/861 20130910
1919458 194 50001-2014 Slovakia ⤷  Subscribe PRODUCT NAME: MERKAPTAMIN; FIRST REGISTRATION: EU/1/13/861, 20130906
>Patent Number >Supplementary Protection Certificate >SPC Country >SPC Expiration >SPC Description

PROCYSBI Market Analysis and Financial Projection Experimental

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.

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Drugs may be covered by multiple patents or regulatory protections. All trademarks and applicant names are the property of their respective owners or licensors. Although great care is taken in the proper and correct provision of this service, thinkBiotech LLC does not accept any responsibility for possible consequences of errors or omissions in the provided data. The data presented herein is for information purposes only. There is no warranty that the data contained herein is error free. thinkBiotech performs no independent verification of facts as provided by public sources nor are attempts made to provide legal or investing advice. Any reliance on data provided herein is done solely at the discretion of the user. Users of this service are advised to seek professional advice and independent confirmation before considering acting on any of the provided information. thinkBiotech LLC reserves the right to amend, extend or withdraw any part or all of the offered service without notice.