{"id":"bmn-333","safety":{"commonSideEffects":[{"rate":"null","effect":"Injection site reactions"},{"rate":"null","effect":"Abdominal pain"},{"rate":"null","effect":"Nausea"}]},"_chembl":{"chemblId":"CHEMBL3654091","moleculeType":"Small molecule","molecularWeight":"990.44"},"_dailymed":null,"mechanism":{"_ai_source":"groq-llama-8b","explanation":"By inhibiting NAGLU, BMN 333 aims to reduce the accumulation of glycosaminoglycans in cells, thereby alleviating the symptoms of MPS IIIB. This approach is based on the understanding that the deficiency of NAGLU leads to the accumulation of these molecules, causing cellular damage and disease progression.","oneSentence":"BMN 333 is a small molecule that targets the enzyme alpha-N-acetylgalactosaminidase (NAGLU), which is deficient in patients with mucopolysaccharidosis type IIIB (MPS IIIB).","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T03:25:10.733Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Mucopolysaccharidosis type IIIB (MPS IIIB)"}]},"trialDetails":[{"nctId":"NCT07441876","phase":"PHASE2, PHASE3","title":"A Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia","status":"NOT_YET_RECRUITING","sponsor":"BioMarin Pharmaceutical","startDate":"2026-02","conditions":"Achondroplasia","enrollment":160}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":2,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":[],"phase":"phase_2","status":"active","brandName":"BMN 333","genericName":"BMN 333","companyName":"BioMarin Pharmaceutical","companyId":"biomarin-pharmaceutical","modality":"Small molecule","firstApprovalDate":"","aiSummary":"BMN 333 is a small molecule that targets the enzyme alpha-N-acetylgalactosaminidase (NAGLU), which is deficient in patients with mucopolysaccharidosis type IIIB (MPS IIIB). Used for Mucopolysaccharidosis type IIIB (MPS IIIB).","enrichmentLevel":3,"visitCount":1,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}