{"id":"bmn-165","safety":{"commonSideEffects":[{"rate":null,"effect":"Elevated liver transaminases"},{"rate":null,"effect":"Immune response/antibody formation"},{"rate":null,"effect":"Injection site reactions"}]},"_chembl":{"chemblId":"CHEMBL4297802","moleculeType":"Enzyme"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"BMN 165 uses an adeno-associated virus (AAV) vector to deliver the galactokinase (GALNS) gene into patient cells, enabling production of the missing or deficient N-acetylgalactosamine-6-sulfatase enzyme. This enzyme restores the ability to break down keratan sulfate and chondroitin-6-sulfate, reducing pathological accumulation of these glycosaminoglycans in tissues and halting or slowing disease progression.","oneSentence":"BMN 165 is a gene therapy that delivers a functional copy of the GALNS gene to restore enzyme activity deficient in mucopolysaccharidosis IVA (Morquio A syndrome).","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:04:31.629Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Mucopolysaccharidosis IVA (Morquio A syndrome)"}]},"trialDetails":[{"nctId":"NCT07477691","phase":"PHASE4","title":"Immune Modulation During Palynziq® Treatment in Adults (IMPALA)","status":"NOT_YET_RECRUITING","sponsor":"BioMarin Pharmaceutical","startDate":"2026-03-31","conditions":"Phenylketonuria","enrollment":12},{"nctId":"NCT05270837","phase":"PHASE3","title":"Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria","status":"ACTIVE_NOT_RECRUITING","sponsor":"BioMarin Pharmaceutical","startDate":"2022-06-17","conditions":"Phenylketonuria (PKU)","enrollment":55},{"nctId":"NCT01889862","phase":"PHASE3","title":"Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2013-07-29","conditions":"Phenylketonuria (PKU)","enrollment":215},{"nctId":"NCT02468570","phase":"","title":"A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2015-08-05","conditions":"Phenylketonuria","enrollment":9},{"nctId":"NCT01560286","phase":"PHASE2","title":"A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2012-05","conditions":"Phenylketonuria","enrollment":24},{"nctId":"NCT01819727","phase":"PHASE3","title":"An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2013-05","conditions":"Phenylketonuria","enrollment":261}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":1,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["rAvPAL-PEG","Pegvaliase"],"phase":"phase_3","status":"active","brandName":"BMN 165","genericName":"BMN 165","companyName":"BioMarin Pharmaceutical","companyId":"biomarin-pharmaceutical","modality":"Small molecule","firstApprovalDate":"","aiSummary":"BMN 165 is a gene therapy that delivers a functional copy of the GALNS gene to restore enzyme activity deficient in mucopolysaccharidosis IVA (Morquio A syndrome). Used for Mucopolysaccharidosis IVA (Morquio A syndrome).","enrichmentLevel":3,"visitCount":1,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}