{"id":"bmn-110-weekly","safety":{"commonSideEffects":[{"rate":null,"effect":"Infusion-related reactions"},{"rate":null,"effect":"Headache"},{"rate":null,"effect":"Nausea"},{"rate":null,"effect":"Fatigue"}]},"_chembl":null,"_fixedAt":"2026-03-30T13:13:41.355388","_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"BMN 110 is designed to replace the deficient lysosomal acid lipase enzyme, which normally breaks down cholesteryl esters and triglycerides within lysosomes. In LAL deficiency (also known as Wolman disease or cholesteryl ester storage disease), accumulation of these lipids causes progressive organ damage. The weekly formulation aims to provide sustained enzyme replacement with less frequent dosing than prior formulations.","oneSentence":"BMN 110 is a recombinant human lysosomal acid lipase (LAL) enzyme replacement therapy that restores deficient enzyme activity in patients with LAL deficiency.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:04:35.072Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Lysosomal acid lipase (LAL) deficiency, including Wolman disease and cholesteryl ester storage disease (CESD)"}]},"_fixedFields":["pubmed(11)"],"trialDetails":[{"nctId":"NCT01415427","phase":"PHASE3","title":"Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2011-07","conditions":"Mucopolysaccharidosis IV A, Morquio A Syndrome, MPS IVA","enrollment":173},{"nctId":"NCT01515956","phase":"PHASE2","title":"Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2011-10","conditions":"Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA","enrollment":15},{"nctId":"NCT01609062","phase":"PHASE2","title":"Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome","status":"TERMINATED","sponsor":"BioMarin Pharmaceutical","startDate":"2012-04","conditions":"Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA","enrollment":25},{"nctId":"NCT01697319","phase":"PHASE2","title":"Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation","status":"TERMINATED","sponsor":"BioMarin Pharmaceutical","startDate":"2012-08","conditions":"Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA","enrollment":16},{"nctId":"NCT01275066","phase":"PHASE3","title":"A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2011-02","conditions":"MPS IV A","enrollment":177},{"nctId":"NCT00884949","phase":"PHASE1, PHASE2","title":"A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2009-04","conditions":"MPS IV A","enrollment":20}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":11,"recentPublications":[{"date":"2025 Aug","pmid":"40411664","title":"rhGALNS Enzyme Stability in Physiological Buffers: Implications for Sustained Release.","journal":"Applied biochemistry and biotechnology"},{"date":"2018 Apr","pmid":"29526614","title":"Safety, immunogenicity, and clinical outcomes in patients with Morquio A syndrome participating in 2 sequential open-label studies of elosulfase alfa enzyme replacement therapy (MOR-002/MOR-100), representing 5 years of treatment.","journal":"Molecular genetics and metabolism"},{"date":"2018 Feb","pmid":"29248359","title":"Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study.","journal":"Molecular genetics and metabolism"},{"date":"2017 Jan","pmid":"27955919","title":"Long-term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study.","journal":"Clinical therapeutics"},{"date":"2016 Dec","pmid":"27855521","title":"Elosulfase alfa (BMN 110) for the treatment of mucopolysaccharidosis IVA (Morquio A Syndrome).","journal":"Expert review of clinical pharmacology"}],"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["N-acetylgalactosamine-6-sulfatase","N-acetylgalactosamine-6-sulfate sulfatase","galactose-6-sulfatase","GALNS","enzyme replacement therapy"],"phase":"phase_3","status":"active","brandName":"BMN 110 - Weekly","genericName":"BMN 110 - Weekly","companyName":"BioMarin Pharmaceutical","companyId":"biomarin-pharmaceutical","modality":"Small molecule","firstApprovalDate":"","aiSummary":"BMN 110 is a recombinant human lysosomal acid lipase (LAL) enzyme replacement therapy that restores deficient enzyme activity in patients with LAL deficiency. Used for Lysosomal acid lipase (LAL) deficiency, including Wolman disease and cholesteryl ester storage disease (CESD).","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}