{"id":"n-acetylgalactosamine-4-sulfatase","safety":{"commonSideEffects":[{"rate":null,"effect":"Infusion-related reactions"},{"rate":null,"effect":"Fever"},{"rate":null,"effect":"Chills"},{"rate":null,"effect":"Headache"},{"rate":null,"effect":"Nausea"}]},"_chembl":{"chemblId":"CHEMBL1201822","moleculeType":"Enzyme"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"The drug is a recombinant human arylsulfatase B enzyme that catalyzes the degradation of glycosaminoglycans (keratan sulfate and chondroitin 6-sulfate) that accumulate in Morquio A syndrome due to GALNS enzyme deficiency. By restoring enzymatic activity, it reduces pathological accumulation of these substrates in tissues and organs, slowing disease progression.","oneSentence":"N-acetylgalactosamine 4-sulfatase is an enzyme replacement therapy that replaces the deficient GALNS enzyme to break down keratan sulfate and chondroitin 6-sulfate in lysosomal storage disease.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T00:50:15.340Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Morquio A syndrome (mucopolysaccharidosis IVA)"}]},"trialDetails":[{"nctId":"NCT01707433","phase":"","title":"Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease","status":"COMPLETED","sponsor":"Children's Hospitals and Clinics of Minnesota","startDate":"2012-10","conditions":"Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI","enrollment":22},{"nctId":"NCT00299000","phase":"PHASE4","title":"A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2006-05","conditions":"Mucopolysaccharidosis VI, Maroteaux-Lamy Syndrome","enrollment":4},{"nctId":"NCT00104234","phase":"PHASE3","title":"Study of rhASB in Patients With Mucopolysaccharidosis VI","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2004-02","conditions":"Mucopolysaccharidosis VI","enrollment":39},{"nctId":"NCT00067470","phase":"PHASE3","title":"Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2003-09","conditions":"Mucopolysaccharidosis VI","enrollment":39},{"nctId":"NCT00048711","phase":"PHASE2","title":"Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2002-03","conditions":"Mucopolysaccharidosis VI","enrollment":""},{"nctId":"NCT00048620","phase":"PHASE1","title":"Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2000-09","conditions":"Mucopolysaccharidosis VI","enrollment":""}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":0,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["rhASB"],"phase":"phase_3","status":"active","brandName":"N-acetylgalactosamine 4-sulfatase","genericName":"N-acetylgalactosamine 4-sulfatase","companyName":"BioMarin Pharmaceutical","companyId":"biomarin-pharmaceutical","modality":"Small molecule","firstApprovalDate":"","aiSummary":"N-acetylgalactosamine 4-sulfatase is an enzyme replacement therapy that replaces the deficient GALNS enzyme to break down keratan sulfate and chondroitin 6-sulfate in lysosomal storage disease. Used for Morquio A syndrome (mucopolysaccharidosis IVA).","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}