CLINICAL TRIALS PROFILE FOR CYSTAGON

All Clinical Trials for CYSTAGON

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.

Clinical Trial Conditions for CYSTAGON

Condition Name

Condition Name for CYSTAGON
Intervention	Trials
Cystinosis	8
Cystic Fibrosis	1
Infantile Neronal Ceroid Lipofuscinosis	1

Condition MeSH

Condition MeSH for CYSTAGON
Intervention	Trials
Cystinosis	8
Fanconi Syndrome	3
Depressive Disorder, Major	1

Clinical Trial Locations for CYSTAGON

Trials by Country

Trials by Country for CYSTAGON
Location	Trials
United States	17
Netherlands	3
France	3
United Kingdom	2
Italy	1

Trials by US State

Trials by US State for CYSTAGON
Location	Trials
California	5
Illinois	4
Georgia	3
Texas	2
Maryland	2

Clinical Trial Progress for CYSTAGON

Clinical Trial Phase

Clinical Trial Phase for CYSTAGON
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	4
Phase 1/Phase 2	3

Clinical Trial Status

Clinical Trial Status for CYSTAGON
Clinical Trial Phase	Trials
Completed	8
Recruiting	2
Terminated	1

Clinical Trial Sponsors for CYSTAGON

Sponsor Name

Sponsor Name for CYSTAGON
Sponsor	Trials
Horizon Pharma USA, Inc.	5
Raptor Pharmaceuticals Inc.	5
Icahn School of Medicine at Mount Sinai	2

Sponsor Type

Sponsor Type for CYSTAGON
Sponsor	Trials
Industry	12
Other	8
NIH	2

CYSTAGON Market Analysis and Financial Projection

Last updated: May 6, 2026

CYSTAGON (cysteamine bitartrate) Clinical Trials Update, Market Analysis, and Projection

What is CYSTAGON and what is its market position today?

CYSTAGON (cysteamine bitartrate) is the long-established oral cysteamine therapy for nephropathic cystinosis. The drug is used to slow kidney damage and is typically deployed chronically, which creates durability in demand even as competitive intensity varies by region.

Market framing (what matters commercially)

• Indication specificity: nephropathic cystinosis is rare; treatable patient pools are stable rather than expansive.
• Chronic therapy: long duration per patient supports recurring demand.
• Competition structure: the core commercial contest is between cysteamine products (including alternative formulations) and access terms, rather than a broad class switch.

Commercial implication: CYSTAGON demand is driven by (1) diagnosis rate and newborn screening uptake, (2) patient adherence and dose persistence, and (3) payer and hospital formulary placement versus alternative cysteamine formulations.

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What is the current clinical-trials update for CYSTAGON?

No actionable, trial-identifying update can be produced from the information available in this session. A complete and accurate “clinical trials update” requires at minimum a trial registry-backed set (e.g., ClinicalTrials.gov identifiers, sponsor, phase, enrollment status, start and primary completion dates, and endpoints) for “CYSTAGON” and/or cysteamine bitartrate assets.

Because that trial-specific record is not available here, this section cannot be filled with verifiable trial facts without risking inaccuracies.

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What endpoints and endpoints’ directionality define cysteamine value?

Even without a new-trial registry feed, cysteamine’s clinical value in nephropathic cystinosis is usually evaluated using a consistent endpoint framework across studies and registries:

1. Corneal cystine burden (typically measured by slit-lamp grading or corneal staining scales)
2. Renal outcomes (e.g., markers of proximal tubular function and renal preservation trajectories)
3. Body cystine burden (biochemical cystine measurements, where collected)
4. Safety and tolerability (gastrointestinal tolerability, dosing limits, discontinuations)

For business and investment screening, the commercial question is whether any new evidence materially changes:

• the proportion of patients who remain on therapy long-term,
• time-to-optimization of dosing regimens,
• or the tolerability profile that affects payer coverage and adherence.

This endpoint logic is stable across the product category; the decision risk sits in whether new trials shift effect size or safety sufficiently to change market share dynamics.

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What is the competitive landscape for oral cysteamine?

CYSTAGON competes within the cysteamine product ecosystem, where the major practical differences are formulation, dosing convenience, taste/administration constraints, and payer preference.

Key competitive drivers

• Formulation and dosing regimen: tablets versus delayed-release or other formulation types can change adherence.
• Access and reimbursement: formulary tiering and prior authorization requirements shape real-world persistence.
• Patient workflow: dosing frequency and administration logistics matter in pediatric chronic use.

Commercial implication: if alternative cysteamine products gain formulary preference in major markets, CYSTAGON growth tends to lag despite stable disease incidence.

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Market analysis: how demand forms for CYSTAGON

A practical market build for CYSTAGON is patient-driven. The demand model should be constructed from three demand layers:

1) Diagnosed patient pool

Demand rises as more nephropathic cystinosis patients are:

• diagnosed earlier,
• treated promptly,
• retained on therapy (adherence and tolerability).

2) Treatment persistence

Since therapy is chronic:

• persistence and dose adherence dominate volume,
• tolerability issues translate into dose interruptions and switching.

3) Pricing and access

Net sales are shaped by:

• reimbursement pressure and discounts,
• tender systems (where applicable),
• formulary placement versus alternatives.

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Market projection: base-case mechanics

A market projection for CYSTAGON in a rare disease should be built from:

• patient growth rate (diagnosis and survival),
• share shift versus competing cysteamine formulations,
• price erosion by region over the projection horizon,
• volume stability due to chronic dosing.

Without trial-by-trial updates and without external pricing and epidemiology inputs in this session, a numerically specific projection cannot be produced without generating unsupported figures.

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Commercial risks that move the projection

Even without registry trial specifics, the main known value drivers for cysteamine products are:

• Formulary preference changes: if alternative cysteamine products become preferred in large payers.
• Adherence/tolerability: persistent use is sensitive to GI tolerability and dosing complexity.
• Pediatric access dynamics: prior authorization and step therapy can shift initiation timing.
• Supply and manufacturing continuity: disruption risk for chronic specialty products affects continuity of therapy.

These risks typically change the market path more than they change total patient pool size.

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What does CYSTAGON’s IP and exclusivity landscape imply for future competition?

A reliable projection depends on whether CYSTAGON faces:

• patent expiry milestones,
• regulatory exclusivity,
• or biosimilar-like equivalent dynamics (not typical here) versus generic competition.

No patent-position facts are provided in this session, and the creation of a factual IP timeline without a cited record would be inaccurate.

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Key Takeaways

• CYSTAGON demand is patient-driven and chronic, with market outcomes dominated by diagnosis timing, persistence, and formulary access versus alternative cysteamine products.
• A registry-backed “clinical trials update” cannot be produced from the available information in this session without introducing trial-identifying errors.
• Projection accuracy hinges on (1) regional reimbursement and formulary share shifts and (2) patient persistence driven by dosing convenience and tolerability.
• Commercial risk is less about effect size changes and more about switching dynamics between cysteamine formulations and access constraints in pediatrics.

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FAQs

1. What is CYSTAGON used for?
   It is used to treat nephropathic cystinosis, aiming to slow organ damage through cystine depletion therapy.

2. What typically drives CYSTAGON sales volume?
   The number of treated patients and long-term treatment persistence, not episodic usage.

3. Who are CYSTAGON’s main commercial competitors?
   Other cysteamine formulations used for nephropathic cystinosis that may have different dosing convenience and formulary status.

4. Do new clinical trials usually change market share for CYSTAGON?
   Market share shifts most when new evidence changes tolerability, adherence, or payer positioning, not only when it repeats known endpoints.

5. What are the biggest risks to market projections for CYSTAGON?
   Formulary access shifts, adherence/tolerability issues, and any therapy continuity disruptions that affect chronic use.

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References

[1] No external sources were provided in this session.