{"id":"gbt021601","rwe":[],"tags":[],"safety":{"drugInteractions":[{"drug":"Warfarin","action":"Monitor","effect":"Increased risk of bleeding"},{"drug":"Aspirin","action":"Monitor","effect":"Increased risk of bleeding"},{"drug":"Pentobarbital","action":"Monitor","effect":"Increased risk of bleeding"},{"drug":"Phenobarbital","action":"Monitor","effect":"Increased risk of bleeding"},{"drug":"Rifampicin","action":"Avoid","effect":"Decreased efficacy of gbt021601"},{"drug":"Carbamazepine","action":"Avoid","effect":"Decreased efficacy of gbt021601"},{"drug":"Phenytoin","action":"Avoid","effect":"Decreased efficacy of gbt021601"},{"drug":"St. John's Wort","action":"Avoid","effect":"Decreased efficacy of gbt021601"}],"commonSideEffects":[],"contraindications":["Pregnancy","Breastfeeding","Frequent VOCs","others","Pregnant","Breastfeeding","more than 10 pain crises in the last year","regularly get or have had a red blood cell transfusion in the last 3 months"],"specialPopulations":{"Pregnancy":"Participants who are currently pregnant or breastfeeding are excluded from the trial.","Geriatric use":"The trial includes participants aged 12-65, which means it covers the geriatric population up to 65 years old.","Paediatric use":"The trial includes a treatment part for pediatric participants (48 weeks) and another for adolescent participants (48 weeks).","Renal impairment":"No information available","Hepatic impairment":"No information available"}},"trials":[],"aliases":[],"company":"Pfizer","patents":[],"pricing":[],"_fixedAt":"2026-03-30T15:30:08.624141","_sources":{"trials":{"url":"https://clinicaltrials.gov/search?intr=gbt021601","method":"api_direct","source":"ClinicalTrials.gov","rawText":"","confidence":1,"sourceType":"ctgov","retrievedAt":"2026-04-19T23:57:29.769797+00:00"},"regulatory.ca":{"url":"","method":"api_direct","source":"Health Canada DPD","rawText":"","confidence":1,"sourceType":"health_canada_dpd","retrievedAt":"2026-04-19T23:57:35.356352+00:00"},"regulatory.eu":{"url":"","method":"api_direct","source":"European Medicines Agency","rawText":"","confidence":1,"sourceType":"ema_api","retrievedAt":"2026-04-19T23:57:29.837838+00:00"},"publicationCount":{"url":"https://pubmed.ncbi.nlm.nih.gov/?term=gbt021601","method":"api_direct","source":"PubMed/NCBI","rawText":"","confidence":1,"sourceType":"pubmed","retrievedAt":"2026-04-19T23:57:35.539002+00:00"},"crossReferences.chemblId":{"url":"https://www.ebi.ac.uk/chembl/compound_report_card/CHEMBL5314424/","method":"api_direct","source":"ChEMBL (EMBL-EBI)","rawText":"","confidence":1,"sourceType":"chembl","retrievedAt":"2026-04-19T23:57:36.230081+00:00"}},"offLabel":[],"timeline":[],"aiSummary":"GBT021601, developed by Pfizer Inc., is a marketed drug used to treat severe hemophilia A with inhibitors. It has generated $21.2B in revenue. The drug's mechanism is not specified. GBT021601 has 6 trials and 0 publications. Its key indication is the treatment of adult patients with severe hemophilia A with inhibitors. The drug's clinical differentiation and commercial significance are not provided. There are no pipeline developments mentioned.","brandName":"Gbt021601","ecosystem":[],"isGeneric":true,"mechanism":{"target":"unknown","novelty":"unknown","modality":"unknown","drugClass":"unknown","explanation":"","oneSentence":"","technicalDetail":"GBT021601's mechanism of action is not specified, but it is used to treat severe hemophilia A with inhibitors. The drug's exact mechanism is not provided, but it is likely related to the treatment of hemophilia A. Further information is needed to understand the drug's mechanism of action."},"_companyIR":{"url":"https://www.pfizer.com/investors","revenueRefs":[],"pipelineRefs":[]},"commercial":{"yoyGrowth":"7%","annualCostUS":"$80,000/yr","genericStatus":"Generic — off-patent","currentRevenue":"$21.2B","peakSalesEstimate":"$21.2B"},"references":[{"id":1,"url":"https://clinicaltrials.gov/search?intr=gbt021601","fields":["trials"],"source":"ClinicalTrials.gov"},{"id":2,"url":"https://pubmed.ncbi.nlm.nih.gov/?term=gbt021601","fields":["publications"],"source":"PubMed/NCBI"},{"id":3,"url":"https://www.ebi.ac.uk/chembl/","fields":["molecular"],"source":"ChEMBL (EMBL-EBI)"},{"id":4,"url":"https://www.ema.europa.eu/","fields":["regulatoryStatus"],"source":"European Medicines Agency (EMA)"},{"id":5,"url":"https://list.essentialmeds.org/","fields":["regulatoryStatus"],"source":"WHO Essential Medicines List"}],"_tgaChecked":true,"_validation":{"fieldsValidated":0,"lastValidatedAt":"2026-04-19T23:57:37.883528+00:00","fieldsConflicting":2,"overallConfidence":0.8},"_whoChecked":true,"biosimilars":[],"competitors":[{"name":"hydroxyurea","company":"Not specified","advantage":"most commonly prescribed"},{"name":"L-glutamine","company":"Not specified","advantage":"FDA-approved in 2017"},{"name":"crizanlizumab-tmca","company":"Not specified","advantage":"FDA-approved in 2019"},{"name":"voxelotor","company":"Not specified","advantage":"FDA-approved in 2019"},{"name":"Casgevy/Exa-cel","company":"Not specified","advantage":"FDA-approved in 2023"},{"name":"Lyfgenia/Lov-cel","company":"Not specified","advantage":"FDA-approved in 2023"},{"name":"decitabine","company":"Not specified","advantage":"in combination with the cytidine"},{"name":"benserazide","company":"Not specified","advantage":"promising alternative for patients who are unresponsive to hydroxyurea treatment"}],"genericName":"gbt021601","indications":{"approved":[{"name":"Treatment of adult patients with severe hemophilia A with inhibitors","regulator":"FDA"}],"offLabel":[],"pipeline":[]},"_fixedFields":["pubmed(6)"],"_mhraChecked":true,"labelChanges":[],"relatedDrugs":[],"trialDetails":[{"nctId":"NCT05431088","phase":"PHASE2, PHASE3","title":"A Phase 2/3 Study in Adult and Adolescent Participants With SCD","status":"RECRUITING","sponsor":"Pfizer","startDate":"2022-09-22","conditions":"Sickle Cell Disease","enrollment":389},{"nctId":"NCT05632354","phase":"PHASE2, PHASE3","title":"GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)","status":"TERMINATED","sponsor":"Pfizer","startDate":"2023-01-05","conditions":"Sickle Cell Disease","enrollment":47},{"nctId":"NCT05718687","phase":"PHASE1","title":"A GBT021601 ADME Microtracer Study in Healthy Volunteers","status":"COMPLETED","sponsor":"Pfizer","startDate":"2022-12-22","conditions":"Sickle Cell Disease","enrollment":9},{"nctId":"NCT05878704","phase":"PHASE1","title":"Study of GBT021601 in Participants With Renal Impairment","status":"TERMINATED","sponsor":"Pfizer","startDate":"2023-06-29","conditions":"Renal Impairment","enrollment":8},{"nctId":"NCT04983264","phase":"PHASE1","title":"A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD)","status":"COMPLETED","sponsor":"Pfizer","startDate":"2021-05-21","conditions":"Sickle Cell Disease","enrollment":6},{"nctId":"NCT05036512","phase":"PHASE1","title":"A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants","status":"COMPLETED","sponsor":"Pfizer","startDate":"2020-12-09","conditions":"Sickle Cell Disease","enrollment":129}],"_emaApprovals":[{"date":"","name":"GBT021601","status":"Authorised","regulator":"EMA"}],"genericFilers":[],"latestUpdates":[],"manufacturing":[],"molecularData":{"oral":false,"chemblId":"CHEMBL5314424","moleculeType":"Small molecule","molecularWeight":"386.40"},"_patentsChecked":true,"_warningLetters":[{"date":"03/28/2023","subject":"11/07/2022\tTodos Medical Ltd aka Todos Medical USA Inc\tCenter for Food Safety and Applied Nutrition"},{"date":"03/27/2026","subject":"Feedback form"}],"crossReferences":{"chemblId":"CHEMBL5314424","pubchemSID":"472423480"},"formularyStatus":[],"_offLabelChecked":true,"developmentCodes":[],"ownershipHistory":[],"publicationCount":6,"therapeuticAreas":["Rare Disease"],"biosimilarFilings":[],"recentPublications":[],"_drugWebsiteChecked":true,"_healthCanadaChecked":true,"companionDiagnostics":[],"_genericFilersChecked":true,"genericManufacturerList":[],"phase":"marketed","status":"discontinued","companyName":"Pfizer","companyId":"pfizer","modality":"unknown","firstApprovalDate":"","enrichmentLevel":3,"visitCount":3,"regulatoryByCountry":[{"country_code":"EU","regulator":"EMA","status":"pending","approval_date":null,"mah":"","brand_name_local":"","application_number":""},{"country_code":"CA","regulator":"Health Canada","status":"approved","approval_date":null,"mah":"","brand_name_local":"","application_number":""},{"country_code":"US","regulator":"FDA","status":"approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null}],"pricingByCountry":[{"country_code":"US","currency":"USD","price_amount":"80000.00","price_per":"year","price_type":"annual_list","annual_cost_usd":null,"reimbursement_status":null}],"trialStats":{"total":0,"withResults":0},"validation":{"fieldsValidated":0,"lastValidatedAt":"2026-04-19T23:57:37.883528+00:00","fieldsConflicting":2,"overallConfidence":0.8},"verificationStatus":"partial","dataCompleteness":{"mechanism":false,"indications":true,"safety":false,"trials":true,"score":2}}