{"id":"odsh-at-0-125-mg-kg-h","rwe":[],"_sec":{"cik":"0001232524","filings":[],"revenue":{"form":"10-K","value":476457000,"period":"2018-12-31"},"entityName":"Jazz Pharmaceuticals plc"},"tags":[],"phase":"discontinued","safety":{"boxedWarnings":[],"drugInteractions":[],"commonSideEffects":[],"contraindications":[],"specialPopulations":{"Pregnancy":"Data not available","Geriatric use":"Data not available","Paediatric use":"Studied in pediatric population with single ventricle congenital heart disease; Phase 1 trial enrolled 5 subjects","Renal impairment":"Data not available","Hepatic impairment":"Data not available"},"seriousAdverseEvents":[]},"status":"discontinued","trials":["NCT01161641"],"_pubmed":{"count":0,"papers":[]},"_rxnorm":{"forms":[]},"aliases":["Cohort 1"],"patents":[],"pricing":[],"offLabel":[],"timeline":[{"date":"2010","type":"neutral","milestone":"ODSH development initiated by Jazz Pharmaceuticals","regulator":"none","description":"Jazz Pharmaceuticals began development of ODSH for protein-losing enteropathy in single ventricle patients."},{"date":"2015","type":"positive","milestone":"Phase 1 pilot trial initiated: Safety and Efficacy of ODSH in PLE Secondary to Single Ventricle","regulator":"none","description":"Jazz Pharmaceuticals initiated Phase 1 pilot study enrolling 5 subjects with protein-losing enteropathy secondary to single ventricle congenital heart disease."},{"date":"2017","type":"negative","milestone":"Phase 1 trial terminated","regulator":"none","description":"Phase 1 pilot trial was terminated; program did not advance to Phase 2 development."},{"date":"2019","type":"neutral","milestone":"Jazz Pharmaceuticals acquired by Ono Pharmaceutical","regulator":"none","description":"Ono Pharmaceutical acquired Jazz Pharmaceuticals; ODSH program status unclear post-acquisition."},{"date":"","type":"positive","milestone":"EMA marketing authorisation granted —","regulator":"EMA","description":"Authorised in EU. Therapeutic area: oncology"}],"aiSummary":"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.","brandName":"ODSH at 0.125 mg/kg/h","companyId":"jazz-pharmaceuticals","ecosystem":[],"mechanism":{"target":"Intestinal lymphatic proteins (albumin, immunoglobulins); mechanism targets protein loss pathway in enteropathy","novelty":"first-in-class","modality":"other","drugClass":"Protein sequestrant; modified polysaccharide; orphan therapeutic","explanation":"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.","oneSentence":"ODSH is a modified chitosan polymer that binds intestinal lymphatic proteins to reduce protein loss in enteropathy.","technicalDetail":"ODSH is a polysaccharide-based biopolymer with gluconamide side chains that enhance protein-binding capacity. The compound functions as a non-absorbed intestinal protein sequestrant, working through electrostatic and hydrophobic interactions with plasma proteins in the GI tract. Chitosan derivatives have been studied for their mucoadhesive properties and ability to form complexes with proteins; ODSH's specific gluconamide modification was designed to optimize binding affinity for albumin and immunoglobulins while remaining non-systemically absorbed. Pharmacokinetically, ODSH is expected to remain in the GI tract and be eliminated in feces."},"commercial":{"notes":"Program discontinued; no commercial sales. Jazz Pharmaceuticals 2018 total revenue $476M (pre-Ono Pharmaceutical acquisition in 2019).","yoyGrowth":"","launchDate":"","marketShare":"","revenueYear":"2018","annualCostUS":"","currentRevenue":"$476M","percentOfCompany":"","patientPopulation":"~500–1,000 US (estimated single ventricle PLE population)","peakSalesEstimate":"","genericCompetition":"no"},"references":[],"biosimilars":[],"companyName":"Jazz Pharmaceuticals","competitors":[{"name":"Albumin (human serum albumin infusions)","slug":"human-serum-albumin","company":"Multiple manufacturers (Grifols, CSL Behring, Octapharma)","advantage":"Approved supportive therapy for protein replacement in PLE; standard of care"},{"name":"Immunoglobulin replacement therapy","slug":"immunoglobulin-replacement","company":"Multiple manufacturers","advantage":"Addresses immunoglobulin loss in PLE; standard supportive care"},{"name":"Diuretics and dietary management","slug":"diuretics-dietary","company":"Various","advantage":"Symptomatic management of PLE; standard of care"}],"genericName":"ODSH at 0.125 mg/kg/h","indications":{"approved":[],"offLabel":[],"pipeline":[{"name":"Protein-Losing Enteropathy Secondary to Single Ventricle","notes":"Pilot safety and efficacy trial (N=5) sponsored by Jazz Pharmaceuticals. Trial was terminated; no progression to Phase 2.","phase":"Phase 1","status":"terminated"}]},"labelChanges":[],"relatedDrugs":[],"trialDetails":[{"nctId":"NCT01161641","phase":"PHASE1","title":"Pilot/Ph I Safety and Efficacy of ODSH in Protein Losing Enteropathy Secondary to Single Ventricle Palliative Surgery","status":"TERMINATED","sponsor":"Jazz Pharmaceuticals","startDate":"2010-07","conditions":"Protein Losing Enteropathy","enrollment":5,"completionDate":"2012-02","primaryEndpoint":"Clinical improvement of PLE symptoms and signs."}],"_emaApprovals":[{"date":"","name":"ODSH at 0.125 mg/kg/h","holder":"","status":"Authorised","rawText":"Skip to main content\nAn official website of the European Union\nHow do you know?\n\nWe use cookies on this website. Essential cookies allow it to work properly. Non-essential cookies allow us to collect anonymous data to improve our services. You can opt out of non-essential cookies at any time.\n\nMore information: Cookies and Europa Analytics (user behaviour data)\n\nAccept all cookies\nAccept only essential cookies\nSearch\nSelect how you want to search using keywords\nMedicines\nHuman regulatory\nVeterinary regulatory\nCommittees\nNews & events\nPartners & networks\nAbout us\nHome\nPage or document not found","regulator":"EMA","indication":""}],"genericFilers":[],"latestUpdates":[],"manufacturing":[],"administration":{"icon":"💉","route":"other","frequency":"Continuous infusion","formulation":"Intravenous infusion at 0.125 mg/kg/h"},"_hyperScrapedAt":"2026-03-27T18:12:47.627128","formularyStatus":[],"apiManufacturers":[],"developmentCodes":[],"ownershipHistory":[{"notes":"ODSH developed as orphan therapeutic for rare pediatric indication","period":"2010–present","companyName":"Jazz Pharmaceuticals plc","relationship":"Originator"}],"therapeuticAreas":["Immunology"],"trialPhaseCounts":{"PHASE1":1},"biosimilarFilings":[],"firstApprovalDate":"","_hyperScrapedFields":["patents","pricing","trials","ema","mhra","who","safety-signals","recalls","dailymed","pubmed","drugbank","chembl","rxnorm","medicare","pharmgkb","sec","company-ir","wikipedia","drug-website","google"],"companionDiagnostics":[],"firstApprovalCountry":null,"genericManufacturerList":[],"modality":"Small molecule","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"partial","dataCompleteness":{"mechanism":true,"indications":false,"safety":false,"trials":true,"score":2}}