Paroxysmal Nocturnal Haemoglobinuria (PNH) (DBCOND0079913)
Identifiers
- Synonyms
- Marchiafava-Micheli Syndrome / PNH - Paroxysmal Nocturnal Hemoglobinuria / Paroxysmal Nocturnal Haemoglobinuria / Paroxysmal nocturnal hemoglobinuria [Marchiafava-Micheli] / Paroxysmal Nocturnal Hemoglobinuria / Paroxysmal nocturnal hemoglobinuria (disorder)
Associated Data
- Indicated Drugs and Targets
Drug Description Targets Danicopan A small molecule complement factor D inhibitor used to treat extravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria.Eculizumab A recombinant humanized monoclonal antibody used to reduce the risk of hemolysis in paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).- Complement C5target
Iptacopan A factor B inhibitor used to treat paroxysmal nocturnal hemoglobinuria.- Cytochrome P450 2C8enzyme
- Cytochrome P450 2D6enzyme
- UDP-glucuronosyltransferase 1A1enzyme
- UDP-glucuronosyltransferase 1A3enzyme
- UDP-glucuronosyltransferase 1A8enzyme
- Complement factor Btarget
- Albumincarrier
- Alpha-1-acid glycoprotein 1carrier
Pegcetacoplan A complement inhibitor indicated in the treatment of adults with paroxysmal nocturnal hemoglobinuria.- Complement C3target
Ravulizumab A monoclonal antibody used to treat paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, and myasthenia gravis.- Complement C5target
- Clinical Trials
Identifier Title Drug(s) Purpose Phase Status NCT04463056 Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH treatment 3 completed NCT05731050 Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) No drug interventions treatment 2 not_yet_recruiting NCT01020188 Study of Global Coagulation Tests in Patients With Paroxysmal Nocturnal Haemoglobinuria No drug interventions Not Available Not Available completed NCT02591862 Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) treatment 2 completed