{"disease":{"id":"transthyretin-mediated-amyloidosis","name":"transthyretin mediated amyloidosis"},"drugs":{"marketed":[{"drug_id":"eplontersen","indication_name":"Polyneuropathy of hereditary transthyretin-mediated amyloidosis","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Wainua","generic_name":"EPLONTERSEN","company_name":"Astrazeneca Ab","drug_phase":"marketed","molecular_target":"","drug_class":"Transthyretin-directed RNA Interaction [EPC]","quality_score":10,"revenue":null,"mechanism":"Wainua works by binding to the transthyretin RNA to prevent the production of the transthyretin protein."},{"drug_id":"acoramidis","indication_name":"cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM)","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Attruby","generic_name":"ACORAMIDIS","company_name":"Bridgebio Pharma","drug_phase":"marketed","molecular_target":"","drug_class":"","quality_score":53,"revenue":null,"mechanism":"Attruby works by stabilizing the transthyretin protein to prevent its misfolding and aggregation into amyloid fibrils."},{"drug_id":"eplontersen","indication_name":"polyneuropathy of hereditary transthyretin-mediated amyloidosis","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Wainua","generic_name":"EPLONTERSEN","company_name":"Astrazeneca Ab","drug_phase":"marketed","molecular_target":"","drug_class":"Transthyretin-directed RNA Interaction [EPC]","quality_score":10,"revenue":null,"mechanism":"Wainua works by binding to the transthyretin RNA to prevent the production of the transthyretin protein."}],"pipeline":[],"offLabel":[],"totalMarketed":3,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT04561518","title":"ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)","phase":"","overall_status":"RECRUITING","enrollment_count":1500,"lead_sponsor_name":"Alnylam Pharmaceuticals","has_results":false},{"nct_id":"NCT07223203","title":"TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy","phase":"PHASE3","overall_status":"RECRUITING","enrollment_count":125,"lead_sponsor_name":"Alnylam Pharmaceuticals","has_results":false},{"nct_id":"NCT05661916","title":"A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":96,"lead_sponsor_name":"Alnylam Pharmaceuticals","has_results":false},{"nct_id":"NCT02797847","title":"A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":80,"lead_sponsor_name":"Alnylam Pharmaceuticals","has_results":false},{"nct_id":"NCT04201418","title":"A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation","phase":"","overall_status":"COMPLETED","enrollment_count":67,"lead_sponsor_name":"Alnylam Pharmaceuticals","has_results":false},{"nct_id":"NCT06527755","title":"A Study to Assess the PK, PD, Safety and Tolerability of Eplontersen in Healthy Chinese Volunteers","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":12,"lead_sponsor_name":"AstraZeneca","has_results":false},{"nct_id":"NCT05040373","title":"Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program","phase":"","overall_status":"RECRUITING","enrollment_count":10,"lead_sponsor_name":"Alnylam Pharmaceuticals","has_results":false},{"nct_id":"NCT05505838","title":"Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy","phase":"","overall_status":"NO_LONGER_AVAILABLE","enrollment_count":null,"lead_sponsor_name":"Alnylam Pharmaceuticals","has_results":false}],"total":8},"guidelines":[],"source":"Drug Landscape verified database"}