CLINICAL TRIALS PROFILE FOR CYSTEAMINE BITARTRATE

All Clinical Trials for CYSTEAMINE BITARTRATE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00028262 ↗	Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 4	2001-02-01	This study will examine the effectiveness of a drug called Cystagon in treating infantile neuronal ceroid lipofuscinosis (INCL), a progressive neurological disease affecting children. At around 11 to 13 months of age, patients develop slowed head growth, mild brain atrophy (wasting), electroencephalographic (EEG) changes and retinal deterioration, with symptoms worsening over time. The disease results from an enzyme deficiency that causes fatty compounds called ceroid to accumulate in cells. In laboratory experiments, Cystagon has helped remove ceroid from cells of patients with INCL. Children with INCL between 6 months and 3 years of age may be eligible for this study. Participants take Cystagon daily by mouth every 6 hours. They are admitted to the NIH Clinical Center for a 4- to 5-day period every 6 months for the following tests and evaluations: - Review of medical history, including a detailed record of seizures, physical examination, blood tests and clinical photographs. For the initial baseline studies, examinations may also be scheduled with pediatric neurology, ophthalmology and anesthesia services. - Magnetic resonance imaging (MRI) of the brain MRI uses a powerful magnet, radio waves, and computers to provide detailed images of the brain without the use of X-rays. The patient lies on a table that slides inside a donut-shaped machine containing a magnetic field. The child requires general anesthesia for the procedure. - Electroretinogram (ERG) measures the function of the retina, the light-sensitive tissue in the back of the eye. To record the flash ERG, a special contact lens is placed on the eye s surface and the eye is stimulated with flashes of light. Infants and very young children require general anesthesia for the procedure. - Visual evoked potential (VEP) measures the function of the visual pathway from the eye to the brain. To record the VEP, five electrodes are placed on the scalp and the eye is stimulated with flashes of light. Infants and very young children must be anesthetized for the procedure. - Electroencephalogram (EEG) measures brain electrical activity, using electrodes placed on the scalp. The test is useful in defining seizures. The child may need to be sedated to keep still during the test. - Skin biopsy A small piece of skin is removed (usually from the upper arm or shoulder) under local anesthetic to grow cells in the laboratory. This procedure is done at the start of the study and is repeated after 1 year if therapy results are promising. Children s condition may improve, stabilize or worsen during this study. Life may be prolonged without significant improvement in quality. The information gained from the study may help scientists develop more potent drugs to treat INCL.
NCT00359684 ↗	Use of Cysteamine in the Treatment of Cystinosis	Recruiting	National Human Genome Research Institute (NHGRI)	Phase 4	1978-07-01	Cystinosis is an inherited disease resulting in poor growth and kidney failure. There is no known cure for cystinosis, although kidney transplantation may help the renal failure and prolong survival. Both the kidney damage and growth failure are thought to be due to the accumulation of the amino acid cystine within the cells of the body. The cystine storage later damages other organs besides the kidneys, including the thyroid gland, pancreas, eyes, and muscle. The drug cysteamine (Cystagon) is an oral medication given to patients with cystinosis prior to kidney transplantation. The drug works by reducing the level of cystine in the white blood cells and muscle tissue. The drug may also decrease levels of cystine in the kidneys and other tissues. This study has several goals: 1. Long-term surveillance of cysteamine (Cystagon) treated patients. 2. Detection of new non-kidney complications of cystinosis. 3. Maintenance of a patient population for genetic testing (mutational analysis) of the cystinosis gene.
NCT00715559 ↗	Cysteamine Therapy for Major Depressive Disorder	Terminated	Icahn School of Medicine at Mount Sinai	N/A	2008-07-01	The purpose of this study is to determine whether cysteamine bitartrate, an FDA-approved drug for a non-psychiatric condition, is safe and effective for the treatment of major depression.
NCT00715559 ↗	Cysteamine Therapy for Major Depressive Disorder	Terminated	Murrough, James, M.D.	N/A	2008-07-01	The purpose of this study is to determine whether cysteamine bitartrate, an FDA-approved drug for a non-psychiatric condition, is safe and effective for the treatment of major depression.

Clinical Trial Conditions for CYSTEAMINE BITARTRATE

Condition Name

Condition Name for CYSTEAMINE BITARTRATE
Intervention	Trials
Cystinosis	8
Nonalcoholic Fatty Liver Disease (NAFLD)	1
Schizoaffective	1
Schizophrenia	1

Condition MeSH

Condition MeSH for CYSTEAMINE BITARTRATE
Intervention	Trials
Cystinosis	8
Fanconi Syndrome	3
Mitochondrial Diseases	2
Schizophrenia	1

Clinical Trial Locations for CYSTEAMINE BITARTRATE

Trials by Country

Trials by Country for CYSTEAMINE BITARTRATE
Location	Trials
United States	36
Netherlands	3
France	3
Belgium	1
Brazil	1

Trials by US State

Trials by US State for CYSTEAMINE BITARTRATE
Location	Trials
California	8
Texas	5
Illinois	5
Georgia	5
Ohio	4

Clinical Trial Progress for CYSTEAMINE BITARTRATE

Clinical Trial Phase

Clinical Trial Phase for CYSTEAMINE BITARTRATE
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	4
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for CYSTEAMINE BITARTRATE
Clinical Trial Phase	Trials
Completed	10
Terminated	3
Recruiting	2

Clinical Trial Sponsors for CYSTEAMINE BITARTRATE

Sponsor Name

Sponsor Name for CYSTEAMINE BITARTRATE
Sponsor	Trials
Horizon Pharma USA, Inc.	7
Raptor Pharmaceuticals Inc.	7
Icahn School of Medicine at Mount Sinai	2

Sponsor Type

Sponsor Type for CYSTEAMINE BITARTRATE
Sponsor	Trials
Industry	16
Other	7
NIH	5

Clinical Trials Update, Market Analysis, and Projection for Cysteamine Bitartrate

Last updated: October 30, 2025

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Introduction

Cysteamine Bitartrate has gained prominence predominantly due to its therapeutic application in treating cystinosis, a rare genetic disorder characterized by abnormal accumulation of cystine within lysosomes. As a promising compound, ongoing clinical trials and evolving market dynamics shape its future trajectory. This report synthesizes recent clinical developments, assesses market opportunities, and projects growth prospects for Cysteamine Bitartrate.

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Clinical Trials Update

Current Clinical Development Status

Cysteamine Bitartrate is primarily under investigation for its established indications and potential new therapeutic uses. The United States Food and Drug Administration (FDA) approved it in 1994 for treating cystinosis, with subsequent amendments optimizing dosing and delivery methods. Recent clinical trials extend beyond cystinosis, exploring its role in other lysosomal storage disorders and oxidative stress-related diseases.

In the last two years, multiple clinical trials have focused on novel formulations and dosage optimization:

• Phase III trials focus on alternative delivery methods, such as extended-release formulations, aimed at improving patient compliance. These formulations are designed to reduce the frequency of dosing while maintaining efficacy.
• Investigational studies examine its potential in neurodegenerative conditions owing to its effects on oxidative stress pathways.

Recent Outcomes and Innovations

A 2022 trial published in the Journal of Pediatric Hematology/Oncology indicated that extended-release cysteamine demonstrated comparable efficacy to conventional formulations in cystinosis patients, with improved tolerability and adherence. These advancements are crucial for chronic treatment management, especially in pediatric populations.

Moreover, explorations into off-label uses—such as for neurodegenerative diseases like Parkinson’s disease—are still at early stages, with preliminary data suggesting antioxidant effects. These trials remain in Phase I or II, reflecting a cautious but ongoing interest in expanding its indications.

Regulatory Environment and Approvals

Globally, cysteamine Bitartrate’s regulatory status remains robust within major markets:

• The FDA approved Cystagon, a cysteamine bitartrate formulation, under orphan drug status, fostering accelerated development pathways.
• The European Medicines Agency (EMA) holds similar approvals, with ongoing post-marketing surveillance.

Moving forward, ongoing clinical data may inform expanded indications and potential label extensions, especially if new formulations demonstrate improved patient outcomes.

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Market Analysis

Market Overview

The global cystinosis treatment market remains niche but is characterized by high unmet needs due to rarity and treatment challenges. According to industry reports, the global orphan drug market, encompassing therapies like cysteamine Bitartrate, is projected to grow at a compound annual growth rate (CAGR) of around 11% from 2022 to 2030 [1].

Key market drivers include:

• Increasing diagnosis of cystinosis, aided by improved genetic testing and awareness campaigns.
• Patient compliance issues with existing treatments, creating demand for extended-release and more tolerable formulations.
• The drug’s orphan status, which provides market exclusivity and incentivizes development.

Competitive Landscape

Major competitors include:

• Cystagon (Manufactured by Horizon Therapeutics): The most widely used immediate-release formulation.
• Procysbi (by Horizon Therapeutics): An extended-release version approved by the FDA in 2017, offering improved dosing convenience.
• Other generics emerging in less-regulated markets.

Horizon Therapeutics dominates the market, controlling a significant share with its consistent pipeline and marketing of Procysbi. However, ongoing trials for novel formulations provide opportunities for market entrants, especially if they address unmet adherence needs.

Market Segmentation

The market segmentation is primarily based on:

• Formulation type: Immediate-release vs. extended-release and novel delivery systems.
• End-user: Pediatric vs. adult patients.
• Geography: North America leads, followed by Europe and Asia-Pacific, where increasing genetic testing and healthcare access expand market reach.

Pricing and Reimbursement Trends

Pricing remains high due to the orphan drug designation, often exceeding $250,000 annually per patient. Reimbursement policies vary, with payers increasingly scrutinizing high-cost therapies. However, orphan drug incentives foster access, and emerging biosimilars or generics could reshape pricing dynamics.

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Market Projection and Future Outlook

Growth Forecast (2023-2030)

Considering current clinical progress and market drivers, the global cystinosis treatment market is expected to expand at a CAGR of 9-11% over the next decade. The expansion will be driven by:

• Introduction of improved formulations, particularly extended-release versions addressing adherence issues.
• Enhanced diagnostic techniques, leading to earlier intervention.
• Expansion into investigational indications, such as neurodegenerative and oxidative stress-related diseases.

By 2030, the market valuation could surpass $3.5 billion, up from approximately $1 billion in 2022 [2].

Key Factors Influencing Growth

• Regulatory approvals for new indications or formulations will catalyze market entry.
• Strategic partnerships among pharmaceutical companies to co-develop or distribute emerging formulations.
• Patient advocacy and awareness campaigns, which influence diagnosis rates and treatment adoption.
• Pricing and reimbursement landscapes, balancing innovation incentives with affordability.

Challenges and Risks

• Market competition from potential biosimilars or alternative therapies can impact pricing and market share.
• Clinical trial failures or delays in expanding indications could hinder growth.
• Pricing pressures and reimbursement constraints in certain markets may limit profitability.

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Key Takeaways

• Clinical progress, especially in extended-release formulations, enhances treatment adherence and may expand the patient base.
• Market growth is robust, driven by increased diagnosis, regulatory support, and unmet needs within rare disease treatments.
• Horizon Therapeutics currently leads but faces emerging competition; strategic R&D and regulatory success are crucial.
• Pricing and reimbursement complexities remain significant hurdles but are manageable through advocacy and regulatory engagement.
• Investors and stakeholders should monitor clinical developments closely, particularly approval timelines for new formulations and indications, to capitalize on growth opportunities.

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FAQs

1. What are the current approved formulations of cysteamine Bitartrate?
   The primary authorized formulations include the immediate-release drug Cystagon and the extended-release Procysbi, both approved by FDA and EMA for cystinosis treatment.

2. Are there ongoing trials for new indications of cysteamine Bitartrate?
   Yes, several early-phase trials investigate its use in neurodegenerative disorders driven by oxidative stress, although these are not yet at advanced stages for regulatory approval.

3. What factors could accelerate market growth?
   Key factors include successful clinical trials demonstrating extended-release formulations' superiority, regulatory approvals for new indications, and increased diagnosis rates due to genetic testing.

4. What challenges does the market for cysteamine Bitartrate face?
   Challenges include high treatment costs, limited patient populations leading to market size constraints, and competition from biosimilars or alternative therapies.

5. How is the pricing of cysteamine Bitartrate expected to evolve?
   Pricing is expected to remain high due to orphan drug incentives, but competitive pressures and biosimilar development could lead to reductions, especially in newer markets and formulations.

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References

[1] Market Research Future. Global Orphan Drug Market Analysis. 2022.
[2] Grand View Research. Cystinosis Treatment Market Size, Share & Trends. 2022.