Guide for the Assessment of Clotting Factor Concentrates

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TABLE 3. FACTOR VIII CONCENTRATES: AFFINITY CHROMATOGRAPHY, OR RECOMBINANTBRAND COMPANY SITE OF MANU-FACTUREAlphanate Grifols Los Angeles,CA, USAPLASMA SOURCE EXPORT/DOMESTICUnited States: paidapheresisBoth Heparin ligandchromatographyFRACTIONATION VIRAL INACTIVATION SA s albCOMMENTSFVIII IU/mg 1TNBP/polysorbate 80 &dry heat, 80 o C, 72 hr≥100 Albumin added, contains VWF;SA w/o alb & VWF = 1000-3000Fanhdi Grifols Barcelona,SpainMonoclate P CSL Behring Kankakee, IL,USAHemofil M AHF BaxterBioScienceLos Angeles,CA, USA1. USA: paid apheresis2. Spain: unpaidrecovered & apheresis3. Czech Republic:recovered & apheresis4. Slovakia, recovered &apheresisUnited States: paidapheresisUnited States: paidapheresis1. Both2.Domestic only3. to CzechRepublic4. to SlovakiaBoth Monoclonal Ab affinitychromatographySame as above Same as above ≥100 Same as aboveBoth Monoclonal Ab affinity & ionexchange chromatographyPasteurizationat 60 o C, 10 hr> 3000 Albumin added, no VWFTNBP/ Octoxynol 9 Approx. 2000 Albumin added, no functionalVWFReplenate Bio ProductsLaboratoryElstree,England, UKUnited States: paidapheresisDomestic Monoclonal Ab affinity & ionexchange chromatographyTNBP/ Triton X 100 > 2000 As aboveAmofil Sanquin OY Sanquin,AmsterdamOctanativ-M Octapharma Stockholm,SwedenAafact Sanquin Amsterdam,NetherlandsGreenMono GreencrossCorpCross Eight M Japanese RedCrossFinland: unpaidrecoveredSwedish unpaidrecovered & apheresisTo Finland Same as above Same as above > 2000 As aboveBoth Same as above Same as above >2000 As aboveNetherlands: unpaid Domestic Same as above Same as above > 2000 As aboveSeoul, Korea Korea: unpaid Domestic Same as above TNBP/ Triton X 100 > 2000 As aboveChitose City,Japan1. Specific activity minus any added albuminRecombinant factor VIII concentrates:BRAND COMPANY SITE OF MANU-FACTUREKogenate FS =KOGENATE Bayer(in EU)Helixate NexGen =Helixate FSBayer Berkeley, CA,USACSLBehringRecombinate rAHF BaxterBioScienceAdvate rAHF PFM BaxterBioscienceMade by Bayer,Berkeley, CAThousand Oaks,CA, USANeuchatel,SwitzerlandReFacto Wyeth Stockholm,Sweden1. Specific activity minus any added albuminJapan: unpaid Domestic Same as above TNBP/ Triton X 100 &nanofiltrationPLASMASOURCENone,recombinantNone,recombinantNone,recombinantNone,recombinantNone,recombinantEXPORT/DOMESTICFRACTIONATION VIRALINACTIVATIONBoth Recombinant: ionexchange &immunoaffinitychromatographyBoth (Identical toKogenate FS)> 2000 As aboveSA s albCOMMENTSFVIII IU/mg 1TNBP/ polysorbate 80 2600 - 6800 Full-length rFVIII; no VWF. Formulated with sucrose.Albumin not added during purification or formulation.TNBP/ polysorbate 80 2600 - 6800 Same as aboveBoth As above > 4000 Full length rFVIII, no functional VWF.: Human serumalbumin added as stabilizerBoth Recombinant TNBP/ polysorbate 80,Triton X 1004000 – 10,000 Full-length rFVIII, no functional VWF: no addition ofhuman- or animal-derived plasma proteins or albumin inthe cell culture, purification or final formulation.Both Recombinant TNBP/ Triton X 100 13,000 B-domain-deleted FVIII, no VWF. No albuminadded in formulation.38 Guide for the Assessment of Clotting Factor Concentrates
BRAND COMPANY SITE OF MANU-FACTUREProplex – T BaxterBioScienceProthroraas ShanghaiRAASTABLE 4: PROTHROMBIN COMPLEX CONCENTRATES (“PCC”; concentrates of prothrombin and factors VII, IX and X)Los Angeles, CA,USABeriplex P/N CSL Behring Marburg,GermanyHaemosolvexFactor IXNationalBioproductsPLASMA SOURCE EXPORT/DOMESTICUnited States: paidapheresisShanghai, China China, paid/unpaidapheresisPinetown, SouthAfricaProfilnine SD Grifols Los Angeles, CA,USAProthrombinex-VFCSLBioplasmaProthromplex-T BaxterBioScienceBebulin VH BaxterBioScienceMelbourne,AustraliaHT DEFIX SNBTS Edinburgh,ScotlandOctaplex Octapharma Vienna, Austria &Lingolsheim,FranceFacnyne GreencrossCorpCofact Sanquin Amsterdam,NetherlandsPPSB-humanSD/Nano 300/600UMAN ComplexD.I.German RedCross NSTOBUnited States, Austria,Germany paid/unpaidFRACTIONATION VIRAL INACTIVATION SA s albCOMMENTSFIX IU/mg 1Both Tricalcium phosphateabsorption, PEGfractionationBoth PEG precipitation,DEAE sephadexExposure to 20% ethanol;dry heat, 60 o C, 144 hrSolvent/ detergent,nanofiltrationBoth DEAE-sephadex Pasteurization at 60 o C,10 hr, & nanofiltration> 8 Heparin added; maximum 3.5 Ufactor VII per IU factor IX3.5 – 5 Contains protein C 700-900 IU per500 IU factor IX; anti-thrombin III,heparin & albumin addedSouth Africa: unpaid Both DEAE-sephadex TNBP/polysorbate 80 0.9 No albumin added;heparin addedUnited States: paidapheresisAustralia, New Zealand,Hong Kong, Malaysia,unpaidVienna, Austria United States, Austria,Czech Republic,Germany, Sweden:mostly paid apheresisVienna, Austria United States: paidapheresisUnited States &Germany: unpaidSweden, Austria,Germany & UnitedStatesBoth Double DEAE cellulosechromatographyBoth DEAE cellulose absorption Dry heat, 80 o C, 72 hrNanofiltrationBoth Ion exchange adsorption Vapor heat, 60 o C for 10hr at 190 mbar, then80 o C for 1 hr at 375 mbarExport toUSABoth Ion exchangechromatographyBoth Ion exchangechromatographiesSeoul, Korea Korea: unpaid Domestic Ion exchangechromatographyNetherlands: unpaid Both DEAE ion exchangechromatographySpringe, Germany Germany: unpaid Domestic DEAE-sephadex,ion exchangechromatographyKedrion Barga,Italy Europe & United States,unpaid & paidKASKADIL LFB France Western Europe,unpaidSolvent/detergent 4 No albumin, heparin orantithrombin III added1 – 5 No albumin addedAnti-thrombin III & heparin addedSame as above Same as above Heparin addedBoth Anion exchange:DEAE-sephadex/sepharose chromatographyDry heat, 80 o C, 72 hr 2 Anti-thrombin III addedTNBP/ polysorbate 80 &nanofiltration1 or more Heparin added,no antithrombin or albumin added,low factor VIIa contentTNBP/ polysorbate 80 @ 6 – 7 No albumin addedTNBP/polysorbate 80 &15 nm nanofiltrationTNBP/ polysorbate 80 &Two nanofiltration steps,50 nm & 15-19 nmTNBP/ polysorbate 80 &Dry heat, 100 o C,30 minAnti-thrombin III added1 Anti-thrombin III & heparin added;no albumin added< 1.6 Anti-thrombin III & heparin added;no albumin added;factor II & factor X titrationBoth Ion exchange chromatography TNBP/ polysorbate 80 ≥0.6 Heparin added, no albuminor anti-thrombin III added1. Specific activity minus any added albuminFEIBA VH BaxterBioscienceNovoSeven =Niastase (in Canada)NovoNordiskTABLE 5: CONCENTRATES PRIMARILY INTENDED FOR USE IN PATIENTS WITH INHIBITORS: Activated Concentrates (Bypassing agents)Vienna, Austria United States, Austria,Czech Republic,Germany, Sweden;mainly paid apheresisCopenhagen,DenmarkBoth Surface-activated PCC,batch-controlledNone Both RecombinantFactor VIIaVapor heat, 60 o C, 10 hr,190mbar then80 o C, 1 hr, 375 mbarNo heparin addedNone Also licensed for use in congenital deficiency offactor VII, in United StatesGuide for the Assessment of Clotting Factor Concentrates 39
Page 1: GUIDE FOR THE ASSESSMENTOF CLOTTING
Page 5 and 6: INTRODUCTIONSelecting therapeutic p
Page 7 and 8: SECTION 1FACTORS AFFECTING THE QUAL
Page 9 and 10: and authorities need to assess each
Page 11 and 12: There are a number of different vir
Page 13 and 14: Treatment Advantages Points to cons
Page 15: measures may have only limited bene
Page 18 and 19: • FDA draft guidelines• FDA ins
Page 20 and 21: Common strengths of U.S. & EU regul
Page 22 and 23: • Audit potential suppliers who m
Page 24 and 25: on HCV reactivity in product sample
Page 26 and 27: Basic requirementsThere are a numbe
Page 28 and 29: treatment, and therefore samples sh
Page 30 and 31: EvaluationSome of the consideration
Page 32 and 33: Figure 2: Production of cryoprecipi
Page 34 and 35: Some measures which should be inclu
Page 37 and 38: APPENDIX 1REGISTRY OF CLOTTING FACT
Page 39 and 40: TABLE 1A: SEROLOGIC TESTS ON INDIVI
Page 41: TABLE 2. FACTOR VIII CONCENTRATES M
Page 45 and 46: TABLE 7: OTHER CLOTTING FACTOR CONC
Page 47 and 48: APPENDIX 2MODEL PRODUCT ASSESSMENT
Page 49: (D) STABILITY AND SHELF LIFEInclude
Page 52 and 53: Nucleic acid testing (NAT): Testing
Page 56: World Federation of Hemophilia1425

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