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ODSH at 0.125 mg/kg/h
ODSH is a modified chitosan polymer that binds intestinal lymphatic proteins to reduce protein loss in enteropathy.
ODSH (N,N'-bis(3-D-gluconamidopropyl)chitosan) is an experimental orphan drug developed by Jazz Pharmaceuticals for protein-losing enteropathy (PLE) secondary to single ventricle congenital heart disease. The compound is a modified chitosan derivative designed to bind and sequester lymphatic fluid proteins in the gastrointestinal tract, reducing protein loss in patients with PLE—a rare and serious complication of Fontan surgery. The program advanced to Phase 1 clinical testing with a small pilot safety and efficacy trial (N=5) sponsored by Jazz, but was subsequently terminated, indicating the program did not progress to later-stage development. No FDA approval was achieved, and the drug remains discontinued with no active commercial presence. The indication represents an ultra-rare pediatric population with limited treatment options, making it a niche orphan therapeutic target.
At a glance
| Generic name | ODSH at 0.125 mg/kg/h |
|---|---|
| Also known as | Cohort 1 |
| Sponsor | Jazz Pharmaceuticals |
| Drug class | Protein sequestrant; modified polysaccharide; orphan therapeutic |
| Target | Intestinal lymphatic proteins (albumin, immunoglobulins); mechanism targets protein loss pathway in enteropathy |
| Modality | Small molecule |
| Therapeutic area | Immunology |
| Phase | discontinued |
Mechanism of action
ODSH (N,N'-bis(3-D-gluconamidopropyl)chitosan) is a synthetic derivative of chitosan, a natural polysaccharide derived from crustacean shells. The drug is designed to work topically within the gastrointestinal tract by binding to serum proteins that leak into the intestinal lumen in patients with protein-losing enteropathy. In the specific context of single ventricle congenital heart disease (particularly post-Fontan procedure), abnormal lymphatic drainage and elevated intestinal lymphatic pressure lead to excessive protein loss through the gut mucosa. ODSH's mechanism is to sequester these proteins in the intestinal lumen, reducing their systemic loss and helping to restore serum albumin and immunoglobulin levels. The drug acts as a local intestinal binder rather than a systemically absorbed therapeutic agent.
Approved indications
Pipeline indications
- Protein-Losing Enteropathy Secondary to Single Ventricle — Phase 1
Common side effects
Key clinical trials
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- ODSH at 0.125 mg/kg/h CI brief — competitive landscape report
- ODSH at 0.125 mg/kg/h updates RSS · CI watch RSS
- Jazz Pharmaceuticals portfolio CI