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Assessment Report 26 April 2019 EMA/CHMP/322871/2019 Committee for Medicinal Products for Human Use (CHMP) Assessment report Doptelet International non-proprietary name: avatrombopag Procedure No. EMEA/H/C/004722/0000 Note Assessment report as adopted by the CHMP with all information of a commercially confidential nature deleted. Official address Domenico Scarlattilaan 6 ● 1083 HS Amsterdam ● The Netherlands Address for visits and deliveries Refer to www.ema.europa.eu/how-to-find-us Send us a question Go to www.ema.europa.eu/contact Telephone +31 (0)88 781 6000 An agency of the European Union © European Medicines Agency, 2019. Reproduction is authorised provided the source is acknowledged. Table of contents 1. Background information on the procedure .............................................. 7 1.1. Submission of the dossier ...................................................................................... 7 1.2. Steps taken for the assessment of the product ......................................................... 8 2. Scientific discussion .............................................................................. 10 2.1. Problem statement ............................................................................................. 10 2.1.1. Disease or condition ......................................................................................... 10 2.1.2. Epidemiology .................................................................................................. 10 2.1.3. Aetiology and pathogenesis .............................................................................. 11 2.1.4. Clinical presentation and diagnosis .................................................................... 11 2.1.5. Management ................................................................................................... 11 2.2. Quality aspects .................................................................................................. 12 2.2.1. Introduction .................................................................................................... 12 2.2.2. Active Substance ............................................................................................. 12 2.2.3. Finished Medicinal Product ................................................................................ 15 2.2.4. Discussion on chemical, pharmaceutical and biological aspects .............................. 18 2.2.5. Conclusions on the chemical, pharmaceutical and biological aspects ...................... 19 2.2.6. Recommendation for future quality development ................................................. 19 2.3. Non-clinical aspects ............................................................................................ 19 2.3.1. Introduction .................................................................................................... 19 2.3.2. Pharmacology ................................................................................................. 19 2.3.3. Pharmacokinetics............................................................................................. 24 2.3.4. Toxicology ...................................................................................................... 29 2.3.5. Ecotoxicity/environmental risk assessment ......................................................... 32 2.3.6. Discussion on non-clinical aspects...................................................................... 34 2.3.7. Conclusion on the non-clinical aspects ................................................................ 38 2.4. Clinical aspects .................................................................................................. 38 2.4.1. Introduction .................................................................................................... 38 2.4.2. Pharmacokinetics............................................................................................. 40 2.4.3. Pharmacodynamics .......................................................................................... 45 2.4.4. Discussion on clinical pharmacology ................................................................... 47 2.4.5. Conclusions on clinical pharmacology ................................................................. 49 2.5. Clinical efficacy .................................................................................................. 50 2.5.1. Dose response studies...................................................................................... 52 2.5.2. Main studies ................................................................................................... 56 2.5.3. Discussion on clinical efficacy ............................................................................ 92 2.5.4. Conclusions on clinical efficacy .......................................................................... 95 2.6. Clinical safety .................................................................................................... 95 2.6.1. Clinical safety .................................................................................................. 95 2.6.2. Discussion on clinical safety ............................................................................ 111 2.6.3. Conclusions on clinical safety .......................................................................... 113 2.7. Risk Management Plan ...................................................................................... 114 2.8. Pharmacovigilance ............................................................................................ 116 2.9. New Active Substance ....................................................................................... 116 2.10. Product information ........................................................................................ 116 Assessment report EMA/CHMP/322871/2019 Page 2/123 2.10.1. User consultation ......................................................................................... 116 2.10.2. Additional monitoring ................................................................................... 116 3. Benefit-Risk Balance............................................................................ 117 3.1. Therapeutic Context ......................................................................................... 117 3.2. Favourable effects ............................................................................................ 118 3.3. Uncertainties and limitations about favourable effects ........................................... 119 3.4. Unfavourable effects ......................................................................................... 119 3.5. Uncertainties and limitations about unfavourable effects ....................................... 120 3.6. Effects Table .................................................................................................... 120 3.7. Benefit-risk assessment and discussion ............................................................... 121 3.7.1. Importance of favourable and unfavourable effects ............................................ 121 3.7.2. Balance of benefits and risks ........................................................................... 121 3.8. Conclusions ..................................................................................................... 122 4. Recommendations ............................................................................... 122 Assessment report EMA/CHMP/322871/2019 Page 3/123 List of abbreviations Quality AP Applicant's Part (or Open Part) of a DMF API Active Pharmaceutical Ingredient ASM Active Substance Manufacturer BDL Below the limit of detection CEP Certificate of Suitability of the EP CMS Concerned Member State CoA Certificate of Analysis CRS Chemical reference substance DL Detection Limit DMF Dimethylformamide DSC Differential scanning Calorimetry DTA Differential thermal analysis ECD Electrochemical detection EDMF European Drug Master File EDQM European Directorate for the Quality of Medicines EP European Pharmacopoeia FID Flame ionisation detection FT-IR Fourrier transmission infra red (spectroscopy) HPLC High performance liquid chromatography IPC In-process control test GC Gas chromatography ICH International conference on harmonisation IR Infra-red KF Karl Fischer LoA Letter of Access LOD Loss on Drying LoD Limit of detection LoQ Limit of Quantitation MA Marketing Authorisation MAH Marketing Authorisation holder MS Mass spectroscopy NIR Near infra-red NLT Not less than NMR Nuclear magnetic resonance NMT Not more than PAR Proven acceptable range PDA Photo diode array PDE Permitted Daily Exposure PVC Polyvinyl chloride PVdC Polyvinyl dichloride Ph.Eur. European Pharmacopoeia QL Quantitation limit QOS Quality Overall Summary RH Relative Humidity RMS Reference member state RoS Route of synthesis RRt Relative retention time Rt Retention time RT Room temperature SAL Sterility assurance level SEM Scanning electron microscopy SM Starting material TLC Thin layer chromatography TG Thermogravimetry TGA Thermo-Gravimetric Analysis UV Ultra violet XRPD X-Ray powder diffraction Assessment report EMA/CHMP/322871/2019 Page 4/123 Non-clinical ADME absorption, distribution, metabolism, and excretion ADP adenosine diphosphate API active pharmaceutical ingredient AST aspartate aminotransferase APD action potential duration ATPase adenosine triphosphatase AUC area under the concentration-time curve AUC(0-24) area under the concentration-time curve from zero time to 24 hours AUC(0-inf)
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