Cysteamine bitartrate - Generic Drug Details

What are the generic sources for cysteamine bitartrate and what is the scope of freedom to operate?

Cysteamine bitartrate is the generic ingredient in two branded drugs marketed by Horizon and Mylan, and is included in three NDAs. There are twelve patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Cysteamine bitartrate has sixty-two patent family members in thirty-four countries.

There are four drug master file entries for cysteamine bitartrate. Two suppliers are listed for this compound.

Summary for cysteamine bitartrate

International Patents:	62
US Patents:	12
Tradenames:	2
Applicants:	2
NDAs:	3
Drug Master File Entries:	4
Finished Product Suppliers / Packagers:	2
Raw Ingredient (Bulk) Api Vendors:	33
Clinical Trials:	15
Patent Applications:	279
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for cysteamine bitartrate
What excipients (inactive ingredients) are in cysteamine bitartrate?	cysteamine bitartrate excipients list
DailyMed Link:	cysteamine bitartrate at DailyMed

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

Constraining patent/regulatory exclusivity:

9,233,077*PED

Dosage:

CAPSULE, DELAYED RELEASE;ORAL

Constraining patent/regulatory exclusivity:

9,233,077*PED

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 cysteamine bitartrate

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

Sponsor	Phase
Nacuity Pharmaceuticals, Inc.	Phase 1/Phase 2
Children's Hospital Medical Center, Cincinnati	Phase 1
Raptor Pharmaceuticals Inc.	Phase 2

See all cysteamine bitartrate clinical trials

Pharmacology for cysteamine bitartrate

Drug Class	Cystine Depleting Agent
Mechanism of Action	Cystine Disulfide Reduction

Paragraph IV (Patent) Challenges for CYSTEAMINE BITARTRATE

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 cysteamine bitartrate

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	203389-001	Apr 30, 2013		RX	Yes	No	9,925,157*PED				Y
Horizon	PROCYSBI	cysteamine bitartrate	CAPSULE, DELAYED RELEASE;ORAL	203389-001	Apr 30, 2013		RX	Yes	No
Horizon	PROCYSBI	cysteamine bitartrate	CAPSULE, DELAYED RELEASE;ORAL	203389-002	Apr 30, 2013		RX	Yes	Yes	9,925,156*PED				Y
Horizon	PROCYSBI	cysteamine bitartrate	GRANULE, DELAYED RELEASE;ORAL	213491-001	Feb 14, 2020		RX	Yes	No	9,198,882*PED				Y
Horizon	PROCYSBI	cysteamine bitartrate	CAPSULE, DELAYED RELEASE;ORAL	203389-001	Apr 30, 2013		RX	Yes	No
Horizon	PROCYSBI	cysteamine bitartrate	GRANULE, DELAYED RELEASE;ORAL	213491-001	Feb 14, 2020		RX	Yes	No	8,026,284*PED				Y

International Patents for cysteamine bitartrate

Country	Patent Number	Title	Estimated Expiration
South Africa	201508783	DELAYED RELEASE CYSTEAMINE BEAD FORMULATION
Canada	2640531	CYSTEAMINE A ENROBAGE ENTERIQUE, CYSTAMINE ET LEURS DERIVES (ENTERICALLY COATED CYSTEAMINE, CYSTAMINE AND DERIVATIVES THEREOF)
Denmark	2535044
Netherlands	300649
Taiwan	201534357	Delayed release cysteamine bead formulation, and methods of making and using same
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)

Supplementary Protection Certificates for cysteamine bitartrate

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1919458	122014000023	Germany		PRODUCT NAME: CYSTEAMIN ODER EIN PHARMAZEUTISCH VERTRAEGLICHES SALZ DAVON; REGISTRATION NO/DATE: EU/1/13/861 20130906
1919458	CR 2014 00013	Denmark		PRODUCT NAME: CYSTEAMIN, HERUNDER MERCAPTAMINBITARTRAT; REG. NO/DATE: EU/1/13/861 20130906
1919458	194 50001-2014	Slovakia		PRODUCT NAME: MERKAPTAMIN; FIRST REGISTRATION: EU/1/13/861, 20130906
1919458	C01919458/01	Switzerland		PRODUCT NAME: MERCAPTAMIN; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 67129 16.08.2019
1919458	C300649	Netherlands		PRODUCT NAME: CYSTEAMINE; REGISTRATION NO/DATE: EU/1/13/861/001-002 20130906
1919458	300649	Netherlands		PRODUCT NAME: CYSTEAMINE; REGISTRATION NO/DATE: EU/1/13/861/001-002 20130910

Cysteamine bitartrate Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Cysteamine Bitartrate

Introduction to Cysteamine Bitartrate

Cysteamine bitartrate is a crucial medication in the treatment of nephropathic cystinosis, a rare genetic disorder characterized by the accumulation of cystine within cells, leading to severe health complications such as renal failure and other systemic issues.

Market Size and Growth

The market for cysteamine bitartrate, primarily driven by its use in treating nephropathic cystinosis, has been growing steadily. As of 2017, the total market size for cystinosis treatments in seven major markets (the United States, EU5 countries, and Japan) was valued at USD 130.46 million. This market is expected to increase at a significant Compound Annual Growth Rate (CAGR) during the forecast period from 2017 to 2028[1].

By 2023, the nephropathic cystinosis treatment market, which includes cysteamine bitartrate, was valued at US$ 332.5 million and is projected to reach over US$ 744.3 million by 2034, growing at a CAGR of 7.4% from 2024 to 2034[5].

Key Market Drivers

Increasing Prevalence and Diagnosis

The total prevalent population of cystinosis in the seven major markets was 2,956 in 2017, with 1,308 of these cases being diagnosed. The expected increase in diagnosed cases due to improved diagnostic techniques and awareness will drive the demand for cysteamine bitartrate[1].

Advanced Healthcare Infrastructure

Regions with superior healthcare infrastructure, such as North America and Europe, contribute significantly to the market growth. These regions have better access to diagnostic tools and treatment options, including cysteamine bitartrate[5].

Research and Development

Increased funding for research and the development of new therapies, such as AVR-RD-04 by Avrobio and ELX-02 by Eloxx Pharmaceuticals, are expected to expand the treatment landscape and attract more patients to cysteamine bitartrate and other emerging treatments[1].

Financial Performance of Cysteamine Bitartrate

Revenue and Sales

The primary revenue generators for cysteamine bitartrate are the oral therapies Procysbi and Cystagon. Procysbi, developed by Raptor Pharmaceutical and now marketed by Horizon Pharma, has been a significant contributor to the market revenue. In Canada, for instance, the financial performance of Procysbi has been substantial, with detailed analyses showing significant profits earned by Horizon from its sale[2].

Pricing and Profitability

The pricing of cysteamine bitartrate formulations, such as Procysbi, is a critical factor in its financial trajectory. The report by Howard Rosen highlights the financial performance and profitability of Procysbi in Canada, including sales volume, price, and costs. The internal rate of return (IRR) calculations indicate the financial feasibility and viability of the product[2].

Market Share and Competition

Cysteamine bitartrate faces competition from other emerging therapies. For example, AVR-RD-04, a lentiviral-based gene therapy, and ELX-02, a ribosomal protein modulator, are expected to compete with Procysbi and Cystagon. However, the established market presence and approval of cysteamine bitartrate formulations ensure they remain major market players[1].

Regional Market Analysis

United States

The United States accounts for the highest market size for cystinosis treatments, including cysteamine bitartrate. In 2017, the market size in the US was USD 86.52 million. The region's advanced healthcare infrastructure and high demand for effective treatments drive this market segment[1].

Europe

Europe is another significant market, driven by higher reported cases of cystinosis, superior healthcare infrastructure, and increased funding for research. The EU5 countries (Germany, France, Italy, Spain, and the UK) are key contributors to the European market[5].

Other Regions

The Asia Pacific region is also expected to grow, although at a slower pace compared to North America and Europe. The market attractiveness in these regions is influenced by the availability of treatment options, including cysteamine bitartrate, and the growing awareness of nephropathic cystinosis[5].

Treatment Availability and Distribution

Medication Availability

Cysteamine bitartrate is available in various forms, including oral capsules (Procysbi and Cystagon) and eye drops (Cystadrops and Cystaran). The distribution channels include hospital pharmacies, retail pharmacies, and online pharmacies, ensuring wide accessibility to patients[5].

Surgical Options

In addition to medication, renal transplantation is a critical treatment option for advanced cases of nephropathic cystinosis. The availability of surgical options in hospitals and specialty surgical centers further supports the market growth[5].

Future Outlook and Emerging Trends

New Therapies

The introduction of new therapies such as AVR-RD-04 and ELX-02 is expected to revolutionize the treatment landscape. These therapies offer different mechanisms of action and potentially more effective treatment options, which could attract a significant patient pool and impact the market dynamics[1].

Market Strategies

Leading market players are focusing on strategies like new product launches, mergers, partnerships, and collaborations to strengthen their market position. Companies such as Amgen Inc., Recordati Rare Diseases, Eloxx Pharmaceuticals Inc., and Viatris Inc. (Mylan) are actively involved in these strategies[5].

Key Takeaways

• The market for cysteamine bitartrate is growing due to increasing prevalence, advanced healthcare infrastructure, and ongoing research.
• Procysbi and Cystagon are the main revenue generators, with significant financial performance in regions like the United States and Canada.
• Emerging therapies like AVR-RD-04 and ELX-02 are expected to compete with existing treatments.
• Regional markets, particularly North America and Europe, drive the market growth due to better healthcare infrastructure and higher demand.
• Distribution channels including hospital pharmacies, retail pharmacies, and online pharmacies ensure wide accessibility.

FAQs

What is the current market size of the nephropathic cystinosis treatment market?

The nephropathic cystinosis treatment market was valued at US$ 332.5 million in 2023 and is projected to reach over US$ 744.3 million by 2034[5].

Which regions are the major contributors to the cystinosis treatment market?

North America and Europe are the major contributors, driven by advanced healthcare infrastructure and higher reported cases of cystinosis[5].

What are the main treatments available for nephropathic cystinosis?

The main treatments include cysteamine bitartrate (Procysbi and Cystagon), Cystadrops and Cystaran for ocular cystinosis, and renal transplantation for advanced cases[1][5].

What are the emerging therapies in the cystinosis treatment market?

Emerging therapies include AVR-RD-04, a lentiviral-based gene therapy, and ELX-02, a ribosomal protein modulator[1].

How does the distribution of cysteamine bitartrate impact its market?

The distribution through hospital pharmacies, retail pharmacies, and online pharmacies ensures wide accessibility to patients, supporting market growth[5].

Sources

1. Global Cystinosis Market Study 2017-2019 & 2020-2028 - Globenewswire
2. Expert Report of Howard Rosen Secretariat - Canada.ca
3. Procysbi capsules - FDA
4. Formulation studies on cysteamine - RGU Repository
5. Nephropathic Cystinosis Treatment Market Size & Share to 2034 - Transparency Market Research