Drug Name:danicopan

Development Code:ACH-4471

CAS:1903768-17-1

Original Research Company:Achillion Pharmaceuticals; AstraZeneca 

2024Year1Month19Day,AstraZeneca announced that Voydeya (danicopan) has been approved by Japan's Ministry of Health, Labour and Welfare (MHLW) as an add-on therapy to standard-of-care complement factor C5 inhibitors Ultomiris (ravulizumab) or Soliris (eculizumab) for the subgroup of patients with paroxysmal nocturnal hemoglobinuria (PNH) who experience significant extravascular hemolysis (EVH) while on C5 inhibitor therapy. This approval was primarily based on positive results from the randomized, double-blind ALPHA pivotal Phase III trial. The trial evaluated the efficacy and safety of Voydeya as an add-on therapy to Ultomiris or Soliris in PNH patients experiencing clinically significant EVH (defined as hemoglobin ≤9.5 g/dL and absolute reticulocyte count ≥120x10^9/L). The results showed，Voydeya Achieved the Primary Endpoint of Hemoglobin Change from Baseline to Week 12 and All Key Secondary Endpoints, including avoidance of transfusions and changes in the Functional Assessment of Chronic Illness Therapy–Fatigue (FACIT-Fatigue) score.

2024Year4Month1Day,VOYDEYA™(danicopan)Has been approved in the United States asravulizumabOreculizumabas an adjunctive therapy for the treatment of adult paroxysmal nocturnal hemoglobinuria(PNH)Extravascular Hemolysis(EVH). The approval of the U.S. Food and Drug Administration is based on the keyALPHA IIIPositive results from the phase trial. The trial lasted12The results of Zhou's preliminary evaluation period were published in The Lancet Haematology. ALPHA IIIThe trial period evaluatedVOYDEYAAsULTOMIRISOrSOLIRISAdditional TreatmentPNHWith clinical significanceEVHThe efficacy and safety of patients. The results showed that from baseline to day12Week,voydeyaReached the primary endpoint of hemoglobin change and all keyThe secondary endpoints, including transfusion avoidance and changes in the Functional Assessment of Chronic Illness Therapy-Fatigue Score. ALPHA IIIResults from the trial period showed,voydeyaOverall, it was well tolerated, with no new safety issues identified. The most common treatment-related adverse events in the trial were headache, nausea, arthralgia, and diarrhea. Earlier,voydeyaHas been approved by the United StatesFDAGranted Breakthrough Therapy Designation and awarded Priority Medicines status by the European Medicines Agency.voydeyaHas also received treatment in the United States, the European Union, and JapanPNHOrphan Drug Designation.

ALPHAIs a key globalIIIPhase clinical trial, aimed at evaluatingVOYDEYAAsC5Add-on Medications for Inhibitor TherapySOLIRISOrULTOMIRISClinically SignificantEVHThePNHEfficacy and safety in patients. In this double-blind, placebo-controlled, multi-dose trialIn the test, patients were enrolled and randomly receivedVOYDEYAOrPlacebo(2:1), while receiving continuous12Week'sSOLIRISOrULTOMIRISTreatment. As of2022Year6Month28Day,63Name Randomized Patient Completion12Pre-specified interim analysis was conducted during the initial evaluation period of the week or after discontinuation of treatment.12Weeks, Placebo+ SOLIRISOrULTOMIRISThe group of patients was transferred.Change toVOYDEYA+  SOLIRISOrULTOMIRISGroup, andVOYDEYA+ SOLIRISOrULTOMIRISThe group of patients continued to receive this treatment.12Week. Complete two treatment periods(24Week)Patients can choose to participate in a two-year long-term extension period, and inSOLIRISOrULTOMIRISContinue to accept outsideVOYDEYATreatment. The open-label period of the study is currently ongoing.

Key Intermediate Synthesis

ParticipateReference WO2017035409