{"id":"alipogene-tiparvovec","rwe":[],"tags":[{"label":"Small Molecule","category":"modality"},{"label":"C10AX10","category":"atc"},{"label":"Active","category":"status"},{"label":"Familial lipoprotein lipase deficiency","category":"indication"},{"label":"uniQure biopharma B.V.","category":"company"}],"phase":"marketed","safety":{"commonSideEffects":[]},"trials":[],"aliases":[],"company":"uniQure biopharma B.V.","patents":[],"pricing":[],"_sources":{"trials":{"url":"https://clinicaltrials.gov/search?intr=ALIPOGENE TIPARVOVEC","method":"api_direct","source":"ClinicalTrials.gov","rawText":"","confidence":1,"sourceType":"ctgov","retrievedAt":"2026-04-20T03:30:58.555279+00:00"},"timeline":{"url":"https://en.wikipedia.org/wiki/Alipogene Tiparvovec","method":"deterministic","source":"Wikipedia","rawText":"","confidence":0.8,"sourceType":"wikipedia","retrievedAt":"2026-04-20T03:31:05.870310+00:00"},"regulatory.ca":{"url":"","method":"api_direct","source":"Health Canada DPD","rawText":"","confidence":1,"sourceType":"health_canada_dpd","retrievedAt":"2026-04-20T03:31:04.430882+00:00"},"regulatory.eu":{"url":"","method":"api_direct","source":"European Medicines Agency","rawText":"","confidence":1,"sourceType":"ema_api","retrievedAt":"2026-04-20T03:30:58.629009+00:00"},"publicationCount":{"url":"https://pubmed.ncbi.nlm.nih.gov/?term=ALIPOGENE TIPARVOVEC","method":"api_direct","source":"PubMed/NCBI","rawText":"","confidence":1,"sourceType":"pubmed","retrievedAt":"2026-04-20T03:31:04.779009+00:00"},"mechanism.target_chembl":{"url":"","method":"api_direct","source":"ChEMBL mechanism: Triglyceride hydrolytic enzyme","rawText":"","confidence":1,"sourceType":"chembl","retrievedAt":"2026-04-20T03:31:05.870247+00:00"},"crossReferences.chemblId":{"url":"https://www.ebi.ac.uk/chembl/compound_report_card/CHEMBL2108304/","method":"api_direct","source":"ChEMBL (EMBL-EBI)","rawText":"","confidence":1,"sourceType":"chembl","retrievedAt":"2026-04-20T03:31:05.521989+00:00"}},"allNames":"glybera","offLabel":[],"synonyms":["alipogene tiparvovec","glybera"],"timeline":[{"date":"2012-10-25","type":"positive","source":"DrugCentral","milestone":"EMA approval (uniQure biopharma B.V.)"}],"aiSummary":"Glybera, also known as Alipogene Tiparvovec, is a gene therapy used to treat Lipoprotein Lipase Deficiency and related conditions such as Familial Hyperlipoproteinemia Type 1 and Familial Hyperchylomicronemia. It works by providing a functional copy of the triglyceride hydrolytic enzyme, which is deficient in these conditions, to help break down triglycerides.","brandName":"Glybera","ecosystem":[{"indication":"Familial lipoprotein lipase deficiency","otherDrugs":[],"globalPrevalence":8000}],"mechanism":{"modality":"Small Molecule","explanation":"","oneSentence":"","technicalDetail":"Glybera is a recombinant adeno-associated virus (rAAV) vector that delivers a functional copy of the lipoprotein lipase gene to muscle cells, allowing them to produce the enzyme and restore normal lipid metabolism."},"_wikipedia":{"url":"https://en.wikipedia.org/wiki/Alipogene_tiparvovec","title":"Alipogene tiparvovec","extract":"Alipogene tiparvovec, sold under the brand name Glybera, is a gene therapy treatment designed to reverse lipoprotein lipase deficiency (LPLD), a rare recessive disorder, due to mutations in LPL, which can cause severe pancreatitis. It was recommended for approval by the European Medicines Agency in July 2012, and approved by the European Commission in November of the same year. It was the first marketing authorisation for a gene therapy treatment in either the European Union or the United States.","wiki_history":"==History==\nGlybera was developed over a period of decades by researchers at the University of British Columbia (UBC). In 1986, Michael R. Hayden and John Kastelein began research at UBC, confirming the hypothesis that LPLD was caused by a gene mutation. Years later, in 2002, Hayden and Colin Ross successfully performed gene therapy on test mice to treat LPLD; their findings were featured on the September 2004 cover of Human Gene Therapy. Ross and Hayden next succeeded in treating cats in the same manner, with the help of Boyce Jones. and by 2015, a total of 27 individuals had been involved in phase III testing. The second phase of testing focused on subjects living along the Saguenay River in Quebec, where LPLD affects people at the highest rate in the world (up to 200 per million) due to the founder effect.\n\n===Price===\nAfter over two years of testing, Glybera was approved in the European Union in 2012. However, after spending millions of euros on Glybera's approval, AMT went bankrupt and its assets were acquired by .—revised to $1 million in 2015,—making it the most expensive medicine in the world at the time. However, replacement therapy, a similar treatment, can cost over $300,000 per year, for life. Afterwards, the three remaining doses in Chiesi's inventory were given away to two patients in Germany and one patient in Italy for  each."},"commercial":{},"references":[{"id":1,"url":"https://drugcentral.org/drugcard/5172","fields":["approvals","synonyms","ATC","PK","indications","contraindications","DDIs","targets","patents","FAERS"],"source":"DrugCentral"},{"id":2,"url":"https://clinicaltrials.gov/search?intr=ALIPOGENE%20TIPARVOVEC","fields":["trials"],"source":"ClinicalTrials.gov"},{"id":3,"url":"https://pubmed.ncbi.nlm.nih.gov/?term=ALIPOGENE TIPARVOVEC","fields":["publications"],"source":"PubMed/NCBI"},{"id":4,"url":"https://en.wikipedia.org/wiki/Alipogene_tiparvovec","fields":["history","overview"],"source":"Wikipedia"}],"_emaChecked":true,"_enrichedAt":"2026-03-30T08:37:17.087336","_validation":{"fieldsValidated":0,"lastValidatedAt":"2026-04-20T03:31:07.163149+00:00","fieldsConflicting":1,"overallConfidence":0.8},"biosimilars":[],"competitors":[{"drugName":"dextrothyroxine","drugSlug":"dextrothyroxine","fdaApproval":"1967-04-14","patentStatus":"Unknown","relationship":"same-class"},{"drugName":"probucol","drugSlug":"probucol","fdaApproval":"1977-02-01","patentStatus":"Unknown","relationship":"same-class"},{"drugName":"ezetimibe","drugSlug":"ezetimibe","fdaApproval":"2002-10-25","patentExpiry":"Apr 30, 2026","patentStatus":"Patent protected","relationship":"same-class"},{"drugName":"mipomersen","drugSlug":"mipomersen","fdaApproval":"2013-01-29","patentExpiry":"Jan 29, 2027","patentStatus":"Patent 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deficiency","source":"DrugCentral","snomedId":238086005,"regulator":"FDA","prevalenceClass":"1-9 / 1 000 000","globalPrevalence":8000,"prevalenceMethod":"orphanet","prevalenceSource":"Orphanet (20301485[PMID])"}],"offLabel":[],"pipeline":[]},"drugCategory":"active","labelChanges":[],"relatedDrugs":[{"drugId":"dextrothyroxine","brandName":"dextrothyroxine","genericName":"dextrothyroxine","approvalYear":"1967","relationship":"same-class"},{"drugId":"probucol","brandName":"probucol","genericName":"probucol","approvalYear":"1977","relationship":"same-class"},{"drugId":"ezetimibe","brandName":"ezetimibe","genericName":"ezetimibe","approvalYear":"2002","relationship":"same-class"},{"drugId":"mipomersen","brandName":"mipomersen","genericName":"mipomersen","approvalYear":"2013","relationship":"same-class"},{"drugId":"lomitapide","brandName":"lomitapide","genericName":"lomitapide","approvalYear":"2012","relationship":"same-class"},{"drugId":"evolocumab","brandName":"evolocumab","genericName":"evolocumab","approvalYear":"2015","relationship":"same-class"},{"drugId":"alirocumab","brandName":"alirocumab","genericName":"alirocumab","approvalYear":"2015","relationship":"same-class"},{"drugId":"bempedoic-acid","brandName":"bempedoic acid","genericName":"bempedoic acid","approvalYear":"2020","relationship":"same-class"},{"drugId":"inclisiran","brandName":"inclisiran","genericName":"inclisiran","approvalYear":"2021","relationship":"same-class"},{"drugId":"evinacumab","brandName":"evinacumab","genericName":"evinacumab","approvalYear":"2021","relationship":"same-class"}],"trialDetails":[{"nctId":"NCT03293810","phase":"","title":"Glybera Registry, Lipoprotein Lipase Deficient (LPLD) Patients","status":"COMPLETED","sponsor":"UniQure Biopharma B.V.","startDate":"2014-06-27","conditions":["Lipoprotein Lipase Deficiency","Familial Hyperlipoproteinemia Type 1","Familial Hyperchylomicronemia"],"enrollment":16,"completionDate":"2023-06"},{"nctId":"NCT02904772","phase":"PHASE2","title":"Alipogene Tiparvovec for the Treatment of LPLD Patients","status":"WITHDRAWN","sponsor":"UniQure Biopharma B.V.","startDate":"2016-10","conditions":["LPL Deficiency"],"enrollment":0,"completionDate":"2020-09"},{"nctId":"NCT01447901","phase":"","title":"Duration of Effect of Alipogene Tiparvovec Treatment, Which Was Administered in Other Studies","status":"TERMINATED","sponsor":"Amsterdam Molecular Therapeutics","startDate":"2011-09","conditions":["Hyperlipoproteinemia Type I"],"enrollment":19,"completionDate":"2012-09"},{"nctId":"NCT01109498","phase":"PHASE2,PHASE3","title":"Safety and Efficacy in LPL-Deficient Subjects of AMT-011, an Adeno-Associated Viral Vector Expressing Human Lipoprotein Lipase [S447X]","status":"UNKNOWN","sponsor":"Amsterdam Molecular Therapeutics","startDate":"2007-08","conditions":["Familial Lipoprotein Lipase Deficiency"],"enrollment":14,"completionDate":"2013-06"},{"nctId":"NCT00891306","phase":"PHASE2,PHASE3","title":"Efficacy and Safety of Human Lipoprotein Lipase (LPL)[S447X] Expressed by an Adeno-Associated Viral Vector in LPL-deficient Subjects","status":"COMPLETED","sponsor":"Amsterdam Molecular Therapeutics","startDate":"2009-02","conditions":["Familial Lipoprotein Lipase Deficiency"],"enrollment":5,"completionDate":"2011-04"}],"_emaApprovals":[{"date":"2012-10-25","status":"Authorised","company":"uniQure biopharma B.V."}],"genericFilers":[],"latestUpdates":[],"manufacturing":[],"crossReferences":{"NDDF":"016036","UNII":"20OK4AFR4Y","INN_ID":"8987","UMLSCUI":"C3653189","chemblId":"CHEMBL2108304","ChEMBL_ID":"CHEMBL2108304","DRUGBANK_ID":"DB13521","PUBCHEM_CID":"5816","MESH_SUPPLEMENTAL_RECORD_UI":"C000721108"},"formularyStatus":[],"_enricherVersion":"v2","developmentCodes":[],"ownershipHistory":[{"period":"present","companyName":"uniQure biopharma B.V.","relationship":"Current Owner"}],"publicationCount":34,"therapeuticAreas":["Metabolic"],"atcClassification":{"source":"DrugCentral","atcCode":"C10AX10","allCodes":["C10AX10"]},"biosimilarFilings":[],"recentPublications":[],"companionDiagnostics":[],"genericManufacturerList":[],"status":"approved","companyName":"uniQure biopharma B.V.","companyId":"uniqure-biopharma-b-v","modality":"Gene therapy","firstApprovalDate":"2012","enrichmentLevel":3,"visitCount":2,"regulatoryByCountry":[{"country_code":"EU","regulator":"EMA","status":"approved","approval_date":"2012-10-25T00:00:00.000Z","mah":"uniQure biopharma B.V.","brand_name_local":"","application_number":""},{"country_code":"EU","regulator":"EMA","status":"withdrawn","approval_date":"2012-10-25T00:00:00.000Z","mah":"uniQure biopharma B.V.","brand_name_local":"Glybera","application_number":"EMEA/H/C/002145"},{"country_code":"CA","regulator":"Health 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