CODING AND REIMBURSEMENT GUIDE

Transcription

1 ODING ND REIMURSEMENT GUIDE Dignosis, ND, nd ID-10-M 1 illing codes for ogultion Fctor IX (Recominnt), lumin Fusion Protein THE FIRST ND ONLY rfix THERPY THT DELIVERS HIGH-LEVEL PROTETION WITH UP TO 14-DY DOSING* * In well-controlled ptients 12 yers nd older, defined s 1 month without spontneous leeding on weekly dose of 40 IU/kg. Plese see Importnt Sfety Informtion on ck pge nd ccompnying full prescriing informtion for IDELVION. ogultion Fctor IX (Recominnt), lumin Fusion Protein

2 IDELVION ODING INFORMTION IDELVION DRUG DMINISTRTION ODES Dignosis nd illing odes for ogultion Fctor IX (Recominnt), lumin Fusion Protein Disclimer: The codes eing provided re from complex nd evolving medicl coding system. The treting physicin is solely responsile for dignosis coding nd determintion of the pproprite ID-10-M codes tht descrie the ptient s condition nd re supported y the medicl record. ll codes re provided for informtionl purposes nd re not n exhustive list. The PT, HPS, 2 ID-10-M, nd ID-10-PS codes provided re sed on M or MS guidelines. The illing prty is solely responsile for coding of services (eg, PT coding). ecuse government nd other third-prty pyor coding requirements chnge periodiclly, plese verify current coding requirements directly with the pyor eing illed. For questions regrding denied clims or ssistnce with pre-certifictions nd the ppels process, plese: ll THE IDELVION RESOURE ENTER T PT dministrtion odes: Outptient Fcilities or Physicin s Office 3 The following PT codes descrie the procedure of IDELVION dministrtion, nd will vry depending on the length of the infusion. PT ode Description Intrvenous push, single or initil sustnce/drug report for infusion of 15 minutes or less Ech dditionl sequentil intrvenous push of the sme sustnce/drug provided in fcility (Fcility use only list seprtely in ddition to code for primry procedure) Intrvenous infusion, for therpy, prophylxis, or dignosis, initil, up to 1 hour ID-10-M Dignosis ode 1 The following ID-10-M code my e used to identify ptient medicl conditions typiclly ssocited with hemophili. ID-10-M ode Description D67 Hereditry Fctor IX deficiency HPS odes 2 The following represents the drug code for IDELVION (ogultion Fctor IX [Recominnt], lumin Fusion Protein). The prescrier is dvised to verify with the pyor or Medicre fiscl intermediry to determine the correct code(s). HPS ode J7202 (Effective 1/1/17) Description Injection, Fctor IX, lumin fusion protein (recominnt), IDELVION, 1 IU IDELVION Ntionl Drug odes (NDs) The olded 0 elow represents the dditionl digit required for 11-digit formtting, which must e used for clims filing. ND Nominl Strength* Interntionl Units (IU) IU of cogultion Fctor IX IU of cogultion Fctor IX IU of cogultion Fctor IX IU of cogultion Fctor IX IU of cogultion Fctor IX *Report the ctul numer of Fctor IX units printed on the vil or crton. Plese see Importnt Sfety Informtion on ck pge nd ccompnying full prescriing informtion for IDELVION. ogultion Fctor IX (Recominnt), lumin Fusion Protein 2 3

3 IDELVION MS-1500 U-04 LIM LIM FORM Form IDELVION MS-1500 U-04 LIM LIM Form FORM DETILS DETILS lim Form Exmple PT. 4 TYPE NTL # OF ILL. MED. RE. # 6 STTEMENT OVERS PERIOD 7 5 FED. TX NO. FROM THROUGH 8 PTIENT NME 10 IRTHDTE 11 SEX DMISSION 12 DTE 13 HR 14 TYPE 15 SR 9 PTIENT DDRESS c d ONDITION ODES 29 DT DHR 17 STT STTE 31 OURRENE 32 OURRENE 33 OURRENE 34 OURRENE 35 OURRENE SPN 36 OURRENE SPN 37 ODE DTE ODE DTE ODE DTE ODE DTE ODE FROM THROUGH ODE FROM THROUGH VLUE ODES VLUE ODES VLUE ODES ODE MOUNT ODE MOUNT ODE MOUNT c D d 42 REV. D. 43 DESRIPTION 44 HPS / RTE / HIPPS ODE 45 SERV. DTE 46 SERV. UNITS 47 TOTL HRGES 48 NON-OVERED HRGES 49 e Field Field 21: 42: Dignosis Revenue or ode Nture of Illness or Injury Enter revenue code tht corresponds to the item or service reported Enter the in ID-10-M Field 43 or dignosis Field 44. code(s) The revenue tht est code descries ssocited the with reson IDELVION the ptient is typiclly is receiving 0636 cre. (drugs requiring detiled coding). However, the revenue code selected is often fcility-dependent, tking into considertion vrious pyor requirements. Field 24: Shded re For Medicid clims, nd for Medicre clims tht will cross over to Medicid s the secondry Field 43: pyor, Description ND informtion in specific formt is required in the shded re ove the line For on which Medicid IDELVION clims, is nd reported for Medicre in Field clims 24D. The tht vrious will cross over Medicid to Medicid plns nd s the Medicre secondry hve pyor, different ND informtion reporting in formts specific for formt this informtion. is required in In Field generl, 43. the provider will need to supply the ND in n 11-digit formt preceded y the The modifier vrious N4 (N4XXXXX-0XXX-XX). Medicid plns nd This Medicre is typiclly hve followed different reporting y the ND formts unit of mesure for this (F2 informtion. [Interntionl generl, Units]) nd the the provider numeric quntity will need of to the supply ND the tht ND ws in dispensed. n 11-digit formt preceded y the modifier N4 (N4XXXXX-0XXX-XX). This is Other typiclly pyors followed my require y the similr ND unit informtion. of mesure heck (F2 with the [Interntionl pyor for specific Units]) requirements. nd the numeric quntity of the ND tht ws dispensed. D Field Field 24D: 46: PT/HPS/Modifier Service Units Enter one HPS illing J7202 unit for IDELVION. every interntionl unit (IU) of IDELVION. (The Modifier exct JW numer Drug of mount units contined discrded, in the not vil dministered is printed on the to ny vil or ptient. crton.) On seprte line, sumit modifier JW plus ny units in single-use vil tht re documented s discrded. Effective Jnury 1, 2017, Medicre clims for discrded drugs or iologicls E mount not dministered Field 67: to Dignosis ny ptient shll e sumitted using the JW modifier. 4 Enter the primry ID-10-M dignosis code in Field 67. onditions Exmples of tht PT coexist coding with options the primry for the IV dignosis infusion/iv or develop push of during IDELVION tretment re: cn e entered in Fields 67 67Q (Intrvenous infusion for therpy, prophylxis or dignosis; initil, up to 1 hour) F (Therpeutic, Field prophylctic 74: Principl or dignostic Procedure injection; intrvenous push, single or initil sustnce/drug). Report When filing for n infusion inptient of 15 clim, minutes enter or ID-10-M less. principl procedure code; for exmple, 30233W1 Trnsfusion of Nonutologous The code selected Fctor is sed IX into on Peripherl the documented Vein, Percutneous service pproch. provided to the ptient PGE OF RETION DTE TOTLS 52 REL. 53 SG. 50 PYER NME 51 HELTH PLN ID 54 PRIOR PYMENTS 55 EST. MOUNT DUE 56 NPI INFO EN. 57 OTHER PRV ID 58 INSURED S NME 59 P.REL 60 INSURED S UNIQUE ID 61 GROUP NME 62 INSURNE GROUP NO Field 44: HPS/Rte/HIPPS ode Enter HPS ode J7202 for IDELVION. Modifier JW Drug mount discrded, not dministered to ny ptient. Effective Jnury 1, 2017, Medicre clims for discrded drugs or iologicls mount not dministered to ny ptient shll e sumitted using the JW modifier. 4 For IDELVION, this is cptured on seprte line in Field 44 s J7202JW, plus ny units in single-use vil tht re documented s discrded in Field 46. D Field 24E: Dignosis Pointer Enter the line numer(s) from Field 21 tht est descries the medicl necessity for the service listed in Field 24D. E Field 24G: Dys or Units Enter one illing unit for every interntionl unit (IU) of IDELVION printed on the vil or crton. E 63 TRETMENT UTHORIZTION ODES 64 DOUMENT ONTROL NUMER 65 EMPLOYER NME D E When filing n outptient clim on seprte line, enter the PT code tht represents the dministrtion procedure performed. F 66 DX 67 D E F G H I J K L M N O P Q 69 DMIT 70 PTIENT 71 PPS DX RESON DX c ODE EI c 74 PRINIPL PROEDURE. OTHER PROEDURE. OTHER PROEDURE 75 ODE DTE ODE DTE ODE DTE 76 TTENDING NPI QUL LST FIRST c. OTHER PROEDURE d. OTHER PROEDURE e. OTHER PROEDURE ODE DTE ODE DTE 77 OPERTING NPI QUL ODE DTE 68 LST FIRST 80 REMRKS OTHER NPI QUL LST FIRST c 79 OTHER NPI QUL d LST FIRST U-04 MS-1450 PPROVED OM NO NU illing ommittee Ntionl Uniform PLESE PRINT OR TYPE THE ERTIFITIONS ON THE REVERSE PPLY TO THIS ILL ND RE MDE PRT HEREOF. PPROVED OM FORM 1500 (02-12) Plese see Importnt Sfety Informtion on ck pge nd ccompnying full prescriing informtion for IDELVION. ogultion Fctor IX (Recominnt), lumin Fusion Protein 48 95

4 IDELVION INPTIENT ODES ID-10-PS (Procedure oding System) 5 The following codes pply to the hospitl inptient setting. Procedure ID-10 ode Description Infusion of Fctor IX 30233W1 Trnsfusion of Nonutologous Fctor IX Into Peripherl Vein, Percutneous pproch Infusion of Fctor IX 30243W1 Trnsfusion of Nonutologous Fctor IX Into entrl Vein, Percutneous pproch Hospitl Revenue odes Revenue codes my pply to IDELVION in the inptient nd outptient settings nd re used in conjunction with drug HPS codes. tegory ode Series Definition Phrmcy 025X Phrmcy Hospitl lood service 038X lood nd lood derivtives Drugs tht require specific identifiction 0636 Revenue code 0636 is necessry for sumitting Medicre clims for lood clotting fctors provided in hospitl ODING ND REIMURSEMENT GUIDE Dignosis, ND, nd ID-10-M 1 illing codes for ogultion Fctor IX (Recominnt), lumin Fusion Protein THE FIRST ND ONLY rfix THERPY THT DELIVERS HIGH-LEVEL PROTETION WITH UP TO 14-DY DOSING* Plese see Importnt Sfety Informtion on ck pge nd ccompnying full prescriing informtion for IDELVION. ogultion Fctor IX (Recominnt), lumin Fusion Protein 6 7

5 IDELVION MS-1500 U-04 LIM LIM FORM IDELVION Form MS-1500 LIM IDELVION Form U-04 LIM Form IDELVION DETILS U-04 L lim Form Exmple lim Form Exmple Field 42: Revenue ode D Field 46: Service Field 42: Units Revenue ode PT. 4 TYPE NTL # OF ILL. MED. RE. # 6 STTEMENT OVERS PERIOD 7 5 FED. TX NO. FROM THROUGH 8 PTIENT NME 10 IRTHDTE 11 SEX DMISSION 12 DTE 13 HR 14 TYPE 15 SR 9 PTIENT DDRESS c d ONDITION ODES 29 DT DHR 17 STT STTE e Enter revenue code tht corresponds to the item or service reported in Field 43 or Field 44. The revenue code ssocited with IDELVION is typiclly 0636 (drugs requiring detiled coding). However, the revenue code selected is often fcility-dependent, tking into considertion vrious pyor requirements. Enter one illing Enter unit for revenue every interntionl code tht corresponds unit (IU) of to IDELVION. the item or service reported in Field 43 or Field 44. The revenue code ssocited (The exct numer with of IDELVION units contined is typiclly in 0636 the vil is (drugs printed requiring on detiled the vil or crton.) coding). However, the revenue code selected is often fcility-dependent, tking into considertion vrious pyor requirements. 31 OURRENE 32 OURRENE 33 OURRENE 34 OURRENE 35 OURRENE SPN 36 OURRENE SPN 37 ODE DTE ODE DTE ODE DTE ODE DTE ODE FROM THROUGH ODE FROM THROUGH E Field 67: Dignosis VLUE ODES VLUE ODES VLUE ODES ODE MOUNT ODE MOUNT ODE MOUNT c D d 42 REV. D. 43 DESRIPTION 44 HPS / RTE / HIPPS ODE 45 SERV. DTE 46 SERV. UNITS 47 TOTL HRGES 48 NON-OVERED HRGES 49 1 Field 43: Description For Medicid clims, nd for Medicre clims tht will cross over to Medicid s the secondry pyor, ND informtion in specific formt is required in Field 43. Enter the primry ID-10-M dignosis Field code 43: in Field Description 67. onditions tht coexist with the primry dignosis or develop during tretment For cn Medicid e entered clims, in nd Fields for 67 67Q. Medicre clims tht will cross over to Medicid s the secondry pyor, ND informtion in specific formt is required in Field The vrious Medicid plns nd Medicre hve different reporting formts for this informtion. In generl, the provider will need to supply the ND in n 11-digit formt preceded y the modifier N4 (N4XXXXX-0XXX-XX). This is typiclly followed y the ND unit of mesure (F2 [Interntionl Units]) nd the numeric quntity of the ND tht ws dispensed. F The vrious Field 74: Medicid Principl plns Procedure nd Medicre hve different reporting formts for this informtion. In generl, the When filing n provider inptient will clim, need enter to supply ID-10-M the ND principl n 11-digit formt procedure code; preceded for exmple, y the 30233W1 modifier N4 Trnsfusion (N4XXXXX-0XXX-XX). of This Nonutologous is Fctor typiclly IX followed into Peripherl y the Vein, ND Percutneous unit of mesure (F2 pproch. [Interntionl Units]) nd the numeric quntity of the ND tht ws dispensed Field 44: HPS/Rte/HIPPS ode Field 44: HPS/Rte/HIPPS ode Enter HPS ode J7202 for IDELVION. Enter HPS ode J7202 for IDELVION PGE OF RETION DTE TOTLS 52 REL. 53 SG. 50 PYER NME 51 HELTH PLN ID 54 PRIOR PYMENTS 55 EST. MOUNT DUE 56 NPI INFO EN. 57 OTHER PRV ID 58 INSURED S NME 59 P.REL 60 INSURED S UNIQUE ID 61 GROUP NME 62 INSURNE GROUP NO Modifier JW Drug mount discrded, not dministered to ny ptient. Effective Jnury 1, 2017, Medicre clims for discrded drugs or iologicls mount not dministered to ny ptient shll e sumitted using the JW modifier. 4 For IDELVION, this is cptured on seprte line in Field 44 s J7202JW, plus ny units in single-use vil tht re documented s discrded in Field 46. Modifier JW Drug mount discrded, not dministered to ny ptient. Effective Jnury 1, 2017, Medicre clims for discrded drugs or iologicls mount not dministered to ny ptient shll e sumitted using the JW modifier. 4 For IDELVION, this is cptured on seprte line in Field 44 s J7202JW, plus ny units in single-use vil tht re documented s discrded in Field 46. E 63 TRETMENT UTHORIZTION ODES 64 DOUMENT ONTROL NUMER 65 EMPLOYER NME D E When filing n outptient clim on seprte line, enter the PT code tht represents the dministrtion procedure performed. D E When filing n outptient clim on seprte line, enter the PT code tht represents the dministrtion procedure performed. F 66 DX 67 D E F G H I J K L M N O P Q 69 DMIT 70 PTIENT 71 PPS DX RESON DX c ODE EI c 74 PRINIPL PROEDURE. OTHER PROEDURE. OTHER PROEDURE 75 ODE DTE ODE DTE ODE DTE 76 TTENDING NPI QUL LST FIRST c. OTHER PROEDURE d. OTHER PROEDURE e. OTHER PROEDURE ODE DTE ODE DTE 77 OPERTING NPI QUL ODE DTE 68 LST FIRST 80 REMRKS OTHER NPI QUL LST FIRST c 79 OTHER NPI QUL d LST FIRST U-04 MS-1450 PPROVED OM NO Ntionl Uniform NU illing ommittee THE ERTIFITIONS ON THE REVERSE PPLY TO THIS ILL ND RE MDE PRT HEREOF. PLESE PRINT OR TYPE PPROVED OM FORM 1500 (02-12) Plese see Importnt Sfety Informtion on ck pge nd ccompnying full prescriing informtion for IDELVION. Plese see Importnt Sfety Informtion on ck pge ccompnying full prescriing informtion for IDELVIO ogultion Fctor IX (Recominnt), lumin Fusion Protein

6 LL NOW FOR REIMURSEMENT SUPPORT IDELVION reimursement nd ccess support is ville free of chrge through My Source SM for IDELVION. LL NOW T My Source SM for IDELVION offers services ove nd eyond reimursement support for your ptients, including: IDELVION Tril Progrm Get your ptients free one-month prescription to see if IDELVION is right for them* o-py svings The My ccess for IDELVION progrm my cover up to $12,000 of ptient out-of-pocket expenses SL ehring ssurnce SM Progrm Helps ptients who rely on IDELVION continue to receive tretment when lpse in coverge occurs *ecuse dosing is sed on vrious ptient trits (such s weight, metolism, etc), there my e cp to the 30-dy supply. Importnt Sfety Informtion IDELVION is indicted in children nd dults with hemophili (congenitl Fctor IX deficiency) for: On-demnd control nd prevention of leeding episodes Periopertive mngement of leeding Routine prophylxis to prevent or reduce the frequency of leeding episodes IDELVION is not indicted for induction of immune tolernce in ptients with hemophili. IDELVION is contrindicted in ptients who hve hd life-thretening hypersensitivity to the product or its components, including hmster proteins. IDELVION is for intrvenous use only. IDELVION cn e self-dministered or dministered y cregiver with trining nd pprovl from helthcre provider or hemophili tretment center. Higher dose per kilogrm ody weight or more frequent dosing my e needed for peditric ptients. Hypersensitivity rections, including nphylxis, re possile. dvise ptients who self-dminister to immeditely report symptoms of hypersensitivity, including ngioedem, chest tightness, hypotension, generlized urticri, wheezing, nd ODING REIMURSEM dyspne. If symptoms occur, discontinue IDELVION nd dminister pproprite tretment. Development of neutrlizing ntiodies (inhiitors) to IDELVION my occur. If expected Fctor IX ctivity plsm levels re not ttined or leeding is not controlled with pproprite dose, perform n ssy to mesure Fctor IX inhiitor concentrtion. Fctor IX ctivity ssy results my vry with the type of ctivted prtil thromoplstin time regent used. Thromoemolism (eg, pulmonry emolism, venous thromosis, nd rteril thromosis) Dignosis, cn occur ND, when nd using ID-10-M 1 illing Fctor IX-contining products. In ddition, nephrotic syndrome hs een reported following immune tolernce (Recominnt), induction lumi in hemophili ptients with Fctor IX inhiitors nd llergic rections to Fctor IX. The most common dverse rection (incidence 1%) reported in clinicl trils ws hedche. Plese see ccompnying full prescriing informtion for IDELVION. References: 1. US Deprtment of Helth & Humn Services, enters for Medicre & Medicid Services. Interntionl lssifiction of Diseses, linicl Modifiction (ID-10-M) ode Tles nd Index. ville t: ccessed Jnury 16, US Deprtment of Helth & Humn Services, enters for Medicre & Medicid Services. HPS Relese odes nd Sets Tle of Drugs. ville t: ccessed Jnury 16, mericn Medicl ssocition. Medicine/hydrtion, therpeutic, prophylctic, dignostic injections nd infusions, nd chemotherpy. In: mericn Medicl ssocition, ed. PT hicgo, IL: mericn Medicl ssocition; 2016: US Deprtment of Helth & Humn Services, enters for Medicre nd Medicid Services. MLN Mtters. ville t: ccessed Jnury 16, US Deprtment of Helth & Humn Services, enters for Medicre &Medicid Services. Interntionl lssifiction of Diseses, Procedure oding System nd Generl Equivlence Mpping (ID-10-PS nd GEMs) ode Tles nd Index. ville t: ID-10-PS-Tles-nd-Index.zip. ccessed Jnury 16, THE FIRST ND ONLY rfi DELIVERS HIGH-LEV WITH UP TO 14-D IDELVION is mnufctured y SL ehring GmH nd distriuted y SL ehring LL. IDELVION is registered trdemrk of SL ehring Recominnt Fcility G. PT is registered trdemrk of the mericn Medicl ssocition. iotherpies for Life nd My ccess re registered trdemrks nd My Source SM nd SL ehring ssurnce SM re service mrks of SL ehring LL SL ehring LL 1020 First venue, PO ox 61501, King of Prussi, P US IDL-0127-MY18 ogultion Fctor IX (Recominnt), lumin Fusion Protein

7 HIGHLIGHTS OF PRESRIING INFORMTION These highlights do not include ll the informtion needed to use IDELVION sfely nd effectively. See full prescriing informtion for IDELVION. IDELVION [ogultion Fctor IX (Recominnt), lumin Fusion Protein] Lyophilized Powder for Solution for Intrvenous Injection Initil U.S. pprovl: INDITIONS ND USGE IDELVION, ogultion Fctor IX (Recominnt), lumin Fusion Protein (rix-fp), recominnt DN-derived cogultion Fctor IX concentrte, is indicted in children nd dults with Hemophili (congenitl Fctor IX deficiency) for: On-demnd tretment nd control of leeding episodes Periopertive mngement of leeding Routine prophylxis to reduce the frequency of leeding episodes Limittions of Use: IDELVION is not indicted for immune tolernce induction in ptients with Hemophili. (1) DOSGE ND DMINISTRTION For intrvenous use fter reconstitution only. Ech vil of IDELVION is leled with the ctul Fctor IX potency in interntionl units (IU). (2.1) One IU of IDELVION per kg ody weight is expected to increse the circulting ctivity of Fctor IX s follows: dolescents nd dults: 1.3 IU/dL per IU/kg (2.1) Peditrics (<12 yers): 1 IU/dL per IU/kg (2.1) dminister intrvenously. Do not exceed infusion rte of 10 ml per minute. (2.3) On-demnd tretment nd control of leeding episodes nd periopertive mngement: Dosge nd durtion of tretment with IDELVION depends on the severity of the Fctor IX deficiency, the loction nd extent of leeding, nd the ptient s clinicl condition, ge nd recovery of Fctor IX. (2.1) Determine the initil dose using the following formul: Required Dose (IU) = ody Weight (kg) x Desired Fctor IX rise (% of norml or IU/dL) x (reciprocl of recovery (IU/kg per IU/dL)) (2.1) djust dose sed on the ptient s clinicl condition nd response. (2.1) Routine prophylxis: Ptients 12 yers of ge: IU/kg ody weight every 7 dys. Ptients who re well-controlled on this regimen my e switched to 14-dy intervl t IU/ kg ody weight. (2.1) Ptients <12 yers of ge: IU/kg ody weight every 7 dys. (2.1) DOSGE FORMS ND STRENGTHS IDELVION is ville s lyophilized powder in single-use vils contining nominlly 250, 500, 1000, 2000, or 3500 IU. (3) ONTRINDITIONS Do not use in ptients who hve hd life-thretening hypersensitivity rections to IDELVION or its components, including hmster proteins. (4) WRNINGS ND PREUTIONS Hypersensitivity rections, including nphylxis, re possile. Should symptoms occur, discontinue IDELVION nd dminister pproprite tretment. (5.1) Development of neutrlizing ntiodies (inhiitors) to IDELVION my occur. If expected Fctor IX plsm recovery in ptient plsm is not ttined, or if leeding is not controlled with n pproprite dose, perform n ssy tht mesures Fctor IX inhiitor concentrtion. (5.2, 5.5) Thromoemolism (e.g., pulmonry emolism, venous thromosis, nd rteril thromosis) my occur when using Fctor IX-contining products. (5.3) Nephrotic syndrome hs een reported following immune tolernce induction with Fctor IX-contining products in hemophili ptients with Fctor IX inhiitors nd history of llergic rections to Fctor IX. (5.4) Fctor IX ctivity ssy results my vry with the type of ctivted prtil thromoplstin time regent used. (5.5) DVERSE RETIONS The most common dverse rection (incidence 1%) reported in clinicl trils ws hedche. (6) To report SUSPETED DVERSE RETIONS, contct SL ehring Phrmcovigilnce Deprtment t or FD t FD-1088 or USE IN SPEIFI POPULTIONS Peditric: Higher dose per kilogrm ody weight or more frequent dosing my e needed. (8.4) See 17 for PTIENT OUNSELING INFORMTION nd FD-pproved ptient leling. Revised: My 2018R FULL PRESRIING INFORMTION: ONTENTS* 1 INDITIONS ND USGE 2 DOSGE ND DMINISTRTION 2.1 Dosge 2.2 Preprtion nd Reconstitution 2.3 dministrtion 3 DOSGE FORMS ND STRENGTHS 4 ONTRINDITIONS 5 WRNINGS ND PREUTIONS 5.1 Hypersensitivity Rections 5.2 Neutrlizing ntiodies 5.3 Thromoemolic omplictions 5.4 Nephrotic Syndrome 5.5 Monitoring Lortory Tests 6 DVERSE RETIONS 6.1 linicl Trils Experience 6.2 Immunogenicity 8 USE IN SPEIFI POPULTIONS 8.1 Pregnncy 8.2 Lcttion 8.4 Peditric Use 8.5 Geritric Use 11 DESRIPTION 12 LINIL PHRMOLOGY 12.1 Mechnism of ction 12.2 Phrmcodynmics 12.3 Phrmcokinetics 13 NONLINIL TOXIOLOGY 13.1 rcinogenesis, Mutgenesis, Impirment of Fertility 14 LINIL STUDIES 15 REFERENES 16 HOW SUPPLIED/STORGE ND HNDLING 17 PTIENT OUNSELING INFORMTION * Sections or susections omitted from the full prescriing informtion re not listed.

8 SL ehring FULL PRESRIING INFORMTION IDELVION ogultion Fctor IX (Recominnt), lumin Fusion Protein (rix-fp) 1 INDITIONS ND USGE IDELVION, ogultion Fctor IX (Recominnt), lumin Fusion Protein (rix-fp), recominnt DN-derived cogultion Fctor IX concentrte, is indicted in children nd dults with Hemophili (congenitl Fctor IX deficiency) for: On-demnd tretment nd control of leeding episodes Periopertive mngement of leeding Routine prophylxis to reduce the frequency of leeding episodes Limittions of Use: IDELVION is not indicted for immune tolernce induction in ptients with Hemophili. 2 DOSGE ND DMINISTRTION For intrvenous use fter reconstitution only. 2.1 Dosge Ech vil of IDELVION contins the recominnt Fctor IX potency in interntionl units (IU) tht is stted on the crton nd vil lel. Dosge nd durtion of tretment with IDELVION depends on the severity of Fctor IX deficiency, the loction nd extent of leeding, nd the ptient s clinicl condition, ge nd recovery of Fctor IX. The clcultion of the required dose of IDELVION is sed on the empiricl finding tht one IU of IDELVION per kg ody weight is expected to increse the circulting level of Fctor IX y 1.3 IU/dL in ptients 12 yers of ge nd y 1 IU/dL in ptients <12 yers of ge. The required dose of IDELVION for tretment of leeding episodes is determined using the following formul: Required Units (IU) = ody Weight (kg) x Desired Fctor IX rise (% of norml or IU/dL) x (reciprocl of recovery (IU/kg per IU/dL)) OR Increse in Fctor IX IU/dL (or % of norml) = Dose (IU) x Recovery (IU/dL per IU/kg)/ ody weight (kg) djust the dose sed on the individul ptient s clinicl condition nd response. On-demnd Tretment nd ontrol of leeding Episodes guide for dosing IDELVION for the on-demnd tretment nd control of leeding episodes is provided in Tle 1. Dosing should im t mintining plsm Fctor IX ctivity level t or ove the plsm levels (in % of norml or IU/dL) outlined in Tle 1. Tle 1. Dosing for On-demnd Tretment nd ontrol of leeding Episodes Type of leeding Episode Minor or Moderte Uncomplicted hemrthrosis, muscle leeding (except iliopsos) or orl leeding Mjor Life or lim thretening hemorrhge, deep muscle leeding, including iliopsos, intrcrnil, retrophryngel irculting Fctor IX ctivity Required [% or (IU/dL)] Frequency of Dosing (hours) Durtion of Therpy (dys)* t lest 1 dy, until leeding stops nd heling is chieved. Single dose should e sufficient for mjority of leeds dys, until leeding stops nd heling is chieved. Mintennce dose weekly. * dpted from the WFH Guidelines for the Mngement of Hemophili. Periopertive Mngement of leeding guide for dosing IDELVION for periopertive mngement of leeding is provided in Tle 2. Tle 2. Dosing for Periopertive Mngement of leeding Type of Surgery Minor (including uncomplicted tooth extrction) Mjor (including intrcrnil, phryngel, retrophryngel, retroperitonel) irculting Fctor IX ctivity Required [% or (IU/dL)] Frequency of Dosing (hours) Durtion of Therpy (dys)* t lest 1 dy, or until heling is chieved. Single dose should e sufficient for mjority of minor surgeries (initil level) * dpted from the WFH Guidelines for the Mngement of Hemophili dys, or until leeding stops nd heling is chieved. Repet dose every hours for the first week or until heling is chieved. Mintennce dose 1-2 times per week. Routine Prophylxis For ptients 12 yers of ge, the recommended dose is IU IDELVION per kg ody weight every 7 dys. Ptients who re well-controlled on this regimen my e switched to 14-dy intervl t IU IDELVION per kg ody weight [see linicl Studies (14)]. For ptients <12 yers of ge, the recommended dose is IU per kg ody weight every 7 dys. djust the dosing regimen sed on individul response. 2.2 Preprtion nd Reconstitution The procedures elow re provided s generl guidelines for the preprtion nd reconstitution of IDELVION. lwys work on clen surfce nd wsh your hnds efore performing the following procedures. Use septic technique during the reconstitution procedure. Reconstitute IDELVION using the diluent (Sterile Wter for Injection) nd trnsfer device (Mix2Vil) provided in the kit. To dminister, you will lso need syringe nd suitle needle (not provided). Ensure the vils of IDELVION nd the Sterile Wter for Injection re t room temperture efore mixing. The reconstitution is performed s descried in Tle 3. Tle 3. IDELVION Reconstitution Instructions 1. Plce the IDELVION vil, diluent vil, nd Mix2Vil trnsfer set on flt surfce. 2. Remove flip cps from the IDELVION nd Sterile Wter for Injection (diluent) vils. 3. Wipe the stoppers with the sterile lcohol sw provided nd llow to dry prior to opening the Mix2Vil trnsfer set pckge. 4. Open the Mix2Vil trnsfer set pckge y peeling wy the lid (Fig. 1). Do not remove the device from the pckge. 5. Plce the diluent vil on flt surfce nd hold the vil tightly. Grip the Mix2Vil trnsfer set together with the cler pckge nd push the plstic spike t the lue end of the Mix2Vil trnsfer set firmly through the center of the stopper of the diluent vil (Fig. 2). 6. refully remove the cler pckge from the Mix2Vil trnsfer set. Do not remove the Mix2Vil trnsfer set or touch the exposed end of the device (Fig. 3). 7. With the IDELVION vil plced firmly on flt surfce, invert the diluent vil with the Mix2Vil trnsfer set ttched nd push the plstic spike of the trnsprent dpter firmly through the center of the stopper of the IDELVION vil (Fig. 4). The diluent will utomticlly trnsfer into the IDELVION vil. 8. With the diluent nd IDELVION vil still ttched to the Mix2Vil trnsfer set, gently swirl the IDELVION vil to ensure tht the powder is fully dissolved (Fig. 5). Do not shke the vil. Fig. 1 Fig. 2 Fig. 3 Fig. 4 Fig. 5

9 9. With one hnd, grsp the IDELVION side of the Mix2Vil trnsfer set nd with the other hnd grsp the lue diluent-side of the Mix2Vil trnsfer set, nd unscrew the set into two pieces (Fig. 6). 10. Drw ir into n empty, sterile syringe. While the IDELVION vil is upright, screw the syringe to the Mix2Vil trnsfer set. Inject ir into the IDELVION vil. 11. While keeping the syringe plunger pressed, invert the system upside down nd drw the concentrte into the syringe y pulling the plunger ck slowly (Fig. 7). 12. Disconnect the filled syringe y unscrewing it from the Mix2Vil trnsfer set (Fig. 8). The reconstituted solution should e cler or yellow to colorless solution. Do not use if prticulte mtter or discolortion is oserved. 13. Use immeditely or within 4 hours of reconstitution. Store reconstituted solution t room temperture. Do not refrigerte. 14. If the dose requires more thn one vil, use seprte, unused Mix2Vil trnsfer set nd Sterile Wter for Injection (diluent) vil for ech product vil. Repet steps to pool the contents of the vils into one syringe. 2.3 dministrtion Fig. 6 Fig. 7 Fig. 8 For intrvenous injection only. Do not mix or dminister IDELVION in the sme tuing or continer with other medicinl products. Visully inspect the finl solution for prticulte mtter nd discolortion prior to dministrtion, nd whenever solution nd continer permit. Do not use if prticulte mtter or discolortion is oserved. ttch the syringe contining the reconstituted IDELVION solution to sterile infusion set nd dminister y intrvenous injection. dpt the infusion rte to the comfort level of ech ptient, not exceeding 10 ml per minute. dminister IDELVION t room temperture nd within 4 hours of reconstitution. Discrd ny unused product. 3 DOSGE FORMS ND STRENGTHS IDELVION is ple yellow to white lyophilized powder supplied in single-use vils contining nominlly 250, 500, 1000, 2000, or 3500 IU of Fctor IX potency. The ctul fctor IX potency is leled on ech vil nd crton. 4 ONTRINDITIONS IDELVION is contrindicted in ptients who hve hd life-thretening hypersensitivity rections to IDELVION, or its components, including hmster proteins [see Wrnings nd Precutions (5.1)]. 5 WRNINGS ND PREUTIONS 5.1 Hypersensitivity Rections Hypersensitivity rections, including nphylxis, re possile. Erly signs of hypersensitivity rections, which cn progress to nphylxis include ngioedem, chest tightness, hypotension, generlized urticri, wheezing, nd dyspne. If hypersensitivity symptoms occur, immeditely discontinue dministrtion nd initite pproprite tretment. IDELVION contins trce mounts of hinese hmster ovry (HO) proteins. Ptients treted with this product my develop hypersensitivity to these non-humn mmmlin proteins. 5.2 Neutrlizing ntiodies The formtion of neutrlizing ntiodies (inhiitors) to Fctor IX my occur. Monitor ll ptients treted with IDELVION for the development of neutrlizing ntiodies (inhiitors) y pproprite clinicl oservtions or lortory tests. Perform n ssy tht mesures Fctor IX inhiitor concentrtion if expected plsm Fctor IX ctivity levels re not ttined, or if the leeding is not controlled with n pproprite dose. Ptients with Fctor IX inhiitors re t n incresed risk of severe hypersensitivity rections or nphylxis if re-exposed to Fctor IX. Evlute ptients experiencing llergic rections for the presence of n inhiitor nd closely monitor ptients with inhiitors for signs nd symptoms of cute hypersensitivity rections, prticulrly during erly phses of exposure to the product [see Wrnings nd Precutions (5.1)]. 5.3 Thromoemolic omplictions Thromoemolism (e.g., pulmonry emolism, venous thromosis, nd rteril thromosis) my occur when using Fctor IX-contining products. ecuse of the potentil risk for thromoemolism with the use of Fctor IX products, monitor for erly signs of thromoemolism nd consumptive cogulopthy when dministering IDELVION to ptients with liver disese, firinolysis, periopertive sttus, or risk fctors for thromoemolic events or disseminted intrvsculr cogultion. 5.4 Nephrotic Syndrome Nephrotic syndrome hs een reported following ttempted immune tolernce induction in hemophili ptients with Fctor IX inhiitors nd history of llergic rections. The sfety nd efficcy of using IDELVION for immune tolernce induction hve not een estlished. 5.5 Monitoring Lortory Tests Monitor Fctor IX plsm levels y one-stge clotting ssy to confirm tht dequte Fctor IX levels hve een chieved nd mintined [see Dosge nd dministrtion (2.1)]. Fctor IX ctivity ssy results my vry with the type of ctivted prtil thromoplstin time (PTT) regent used in the ssy system. 1 For exmple, kolin-sed PTT regents long with other regents designed to exhiit low responsiveness to lupus nticogulnt 2 hve een shown to result in pproximtely 50% lower thn expected recovery sed on leled potency. onsistent with similr findings for other recominnt Fctor IX products, overestimtion of Fctor IX ctivity in spiked smples of IDELVION (men overestimtion 32%) occurred t low Fctor IX levels with commonly used PTT regents. 3 Monitor ptients for the development of inhiitors if expected Fctor IX ctivity plsm levels re not ttined, or if leeding is not controlled with the recommended dose of IDELVION. ssys used to determine if Fctor IX inhiitor is present should e titered in ethesd Units (Us). 6 DVERSE RETIONS The most common dverse rection (incidence 1%) reported in clinicl trils ws hedche. 6.1 linicl Trils Experience ecuse clinicl studies re conducted under widely vrying conditions, dverse rection rtes oserved in the clinicl trils of drug cnnot e directly compred to rtes in the clinicl trils of nother drug nd my not reflect the rtes oserved in clinicl prctice. In five multicenter, prospective, open-lel clinicl trils with IDELVION, 111 previously treted ptients (PTPs; exposed to Fctor IX-contining product for 100 exposure dys) received t lest one infusion of IDELVION s prt of on-demnd tretment of leeding episodes, periopertive mngement of mjor nd minor surgicl, dentl, or other invsive procedures, routine prophylxis, or phrmcokinetic evlution. Twenty-seven children were <12 yers of ge, 9 dolescents were 12 to <18 yers of ge, nd 75 dults were 18 to 65 yers of ge. totl of 8,274 injections were dministered over medin of 600 dys (rnge: 25 to 1,288 dys). dverse rections tht occurred in >0.5% of sujects re listed in Tle 4. Tle 4. Summry of dverse Rections MedDR Stndrd System Orgn lss Nervous system disorders Immune system disorders Skin nd sucutneous tissue disorders dverse Rection Numer of sujects n (%), (N=111) Hedche 2 (1.8) Dizziness 1 (0.9) Hypersensitivity 1 (0.9) Rsh 1 (0.9) Eczem 1 (0.9) 6.2 Immunogenicity ll sujects were monitored for inhiitory nd inding ntiodies to rix-fp (specificlly rfix, pdfix nd lumin), nd inding ntiodies to HO host cell proteins t the following time points: t screening, t 2-4 weeks, 12 weeks following the first infusion of IDELVION, every 3 months therefter (every 6 months for inding ntiodies in children). No sujects developed ntiodies to Fctor IX, or non-neutrlizing ntiodies ginst fctor IX, lumin nd HO protein t ny of the time points following infusion of IDELVION. The detection of ntiody formtion is highly dependent on the sensitivity nd specificity of the ssy. dditionlly, the oserved incidence of ntiody (including neutrlizing ntiody) positivity in n ssy my e influenced y severl fctors including ssy methodology, smple hndling, timing of smple collection, concomitnt medictions, nd underlying disese. For these resons, it my e misleding to compre the incidence of ntiodies to IDELVION in the studies descried ove with the incidence of ntiodies in other studies or to other products.

10 8 USE IN SPEIFI POPULTIONS 8.1 Pregnncy Risk Summry There re no dt with IDELVION use in pregnnt women to inform on drug-ssocited risk. niml reproduction studies hve not een conducted using IDELVION. It is not known whether IDELVION cn cuse fetl hrm or ffect reproduction cpcity when dministered to pregnnt womn. IDELVION should e given to pregnnt womn only if clerly needed. In the U.S. generl popultion, the estimted ckground risk of mjor irth defects nd miscrrige in cliniclly recognized pregnncies is 2-4% nd 15-20%, respectively. 8.2 Lcttion Risk Summry There is no informtion regrding the excretion of IDELVION in humn milk, the effect on the restfed infnt, or the effects on milk production. The developmentl nd helth enefits of restfeeding should e considered long with the mother s clinicl need for IDELVION nd ny potentil dverse effects on the restfed infnt from IDELVION or from the underlying mternl condition. 8.4 Peditric Use In clinicl studies tht included 34 sujects <18 yers old, the prophylctic dministrtion with IDELVION every 7 dys ws successful in prevention of spontneous leeding episodes requiring tretment [see linicl Studies (14)]. Of these, 7 sujects were 12 nd <18 yers old; 5 of these sujects switched to 10- or 14-dy prophylctic dministrtion with IDELVION. There were no pprent differences in the sfety profile in sujects <18 yers s compred to dults [see dverse Rections (6.1)]. ompred to dults, incrementl rix-fp recovery ppered to e slightly lower nd ody weight-djusted clernce ppered to e higher. hildren my hve higher Fctor IX ody weight-djusted clernce, shorter hlf-life, nd lower recovery. Higher dose per kilogrm ody weight or more frequent dosing my e needed in these ptients [see Dosge nd dministrtion (2.1) nd linicl Phrmcology (12.3)]. 8.5 Geritric Use linicl studies of IDELVION did not include sujects over 65 yers to determine whether or not they respond differently from younger sujects. 11 DESRIPTION IDELVION, ogultion Fctor IX (Recominnt), lumin Fusion Protein (rix-fp) is sterile, non-pyrogenic, lyophilized powder to e reconstituted with sterile Wter for Injection (swfi) for intrvenous dministrtion. IDELVION is ville in single-use vils contining nominlly 250, 500, 1000, 2000, or 3500 IU of Fctor IX formulted with sodium citrte, polysorte 80, mnnitol nd sucrose. The ctul mount of Fctor IX ctivity in IU is leled on ech vil. fter reconstitution of the lyophilized powder, ll dosge strengths yield cler, yellow to colorless solution. IDELVION contins no preservtives. The ctive ingredient in IDELVION, recominnt humn cogultion Fctor IX lumin fusion protein, is purified protein produced y recominnt DN technology. It is generted y the genetic fusion of recominnt lumin to recominnt cogultion Fctor IX. The genetic fusion of the cdn of humn lumin to the cdn of humn cogultion Fctor IX enles the gene product to e expressed s single recominnt protein designted s rix-fp. The Fctor IX portion of IDELVION is identicl to the Thr148 llelic form of humn plsm-derived Fctor IX. The clevle linker etween the Fctor IX nd lumin moieties is derived from the endogenous ctivtion peptide in ntive Fctor IX. rix-fp remins intct in the circultion until Fctor IX is ctivted, whereupon lumin is cleved from Fctor IX, relesing ctivted Fctor IX (FIX) when it is needed for cogultion. IDELVION is mnufctured without the ddition of proteins derived from humn or niml source mterils. IDELVION is glycoprotein consisting of 1018 mino cids secreted y geneticlly engineered hinese hmster ovry (HO) cell line. The HO cell line secretes rix-fp into defined cell culture medium nd the rix-fp protein is purified y process tht does not require the use of monoclonl ntiody regent. The mnufcturing process incorportes three vlidted virus clernce steps, including virus inctivtion y solvent/ detergent tretment nd virus removl y filtrtion. The potency expressed in Interntionl Units is determined using n in vitro PTT-sed one-stge clotting ssy ginst SL ehring s mnufcturing reference stndrd. This internl potency stndrd hs een clirted ginst the World Helth Orgniztion (WHO) Interntionl Stndrd for Fctor IX concentrte y one-stge clotting ssy using synthetic silic nd synthetic phospholipid-sed regents. 12 LINIL PHRMOLOGY 12.1 Mechnism of ction IDELVION is recominnt protein tht temporrily replces the missing cogultion Fctor IX needed for effective hemostsis. IDELVION is comprised of geneticlly fused recominnt cogultion Fctor IX nd recominnt lumin. Fusion with recominnt lumin extends the hlf-life of Fctor IX [see Description (11) nd linicl Phrmcology (12.3)] Phrmcodynmics The dministrtion of IDELVION increses plsm levels of Fctor IX nd cn temporrily correct the cogultion defect in ptients Phrmcokinetics dults 18 yers The phrmcokinetic (PK) profiles of IDELVION were evluted following n intrvenous injection of single dose of 25, 50 or 75 IU/kg. The PK prmeters were sed on plsm Fctor IX ctivity mesured y the one-stge clotting ssy. lood smples for PK nlysis were collected prior to dosing nd up to 336 hours (14 dys) fter dosing. Tle 5 provides the phrmcokinetic prmeters following single 25 IU/kg, 50 IU/kg, or 75 IU/kg dose of IDELVION. Tle 5. Phrmcokinetic Prmeters (rithmetic Men, V%) Following Single 25 IU/kg, 50 IU/kg, or 75 IU/kg Dose of IDELVION PK Prmeters rix-fp 25 (IU/kg) (N=7) rix-fp 50 (IU/kg) (N=47) rix-fp 75 (IU/kg) (N=8) IR (IU/dL)/(IU/kg) 1.65 (11) 1.30 (24) 1.08 (20) mx (IU/dL) 41.1 (13) 66.6 (27) 82.0 (20) U 0-inf (h*iu/dl) 4658 (36) 7482 (28) 9345 (20) t 1/2 (hours) 118 (38) 104 (25) 104 (18) MRT (hours) 153 (24) 143 (23) 145 (14) L (ml/h/kg) 0.57 (31) 0.73 (27) 0.84 (20) Vss (dl/kg) 0.86 (32) 1.02 (28) 1.20 (23) Time to 1% Fctor IX 22 (25) 23 (19) 25 (13) ctivity (dys) * Time to 3% Fctor IX 14 (19) 17 (19) 18 (13) ctivity (dys) * Time to 5% Fctor IX ctivity (dys) * 10 (25) 13 (20) 15 (13) * = Estimted time to men Fctor IX ctivity mintined ove the pre-specified percent (%) IR = incrementl recovery recorded 30 minutes fter injection; U = re under the Fctor IX ctivity time curve; t 1/2 = hlf-life; MRT = men residence time; L = ody weight djusted clernce; Vss = ody weight djusted volume of distriution t stedy-stte. IDELVION PK prmeters following single or repet dosing for up to 30 weeks were similr. Sujects <18 yers Phrmcokinetics prmeters of IDELVION were evluted in 5 dolescents (12 to <18 yers of ge) nd 27 children (0 to <12 yers of ge) in open-lel, multi-center studies following 50 IU/kg intrvenous injection of IDELVION. The PK smples were collected prior to dosing nd t multiple time points up to 336 hours (14 dys) fter dosing. Tle 6 summrizes the PK prmeters clculted from the peditric dt of 32 sujects, 0 to <18 yers of ge. The prmeters estimted were sed on the plsm Fctor IX ctivity over time profile. ompred to dults, incrementl recovery is lower (15% to 27%) nd ody weight-djusted clernce is higher (45% to 62%) in children. Tle 6. Phrmcokinetic Prmeters (rithmetic Men, V%) of IDELVION Following Single 50 IU/kg Dose PK Prmeters 0 to <6 yers (N=12) 6 to <12 yers (N=15) 12 to <18 yers (N=5) IR (IU/dL)/(IU/kg) 0.95 (22) 1.06 (23) 1.11 (28) mx (IU/dL) 48.3 (19) 52.9 (23) 55.3 (28) U 0-inf (h*iu/dl) 4583 (33) 5123 (31) 5347 (48) t 1/2 (hours) 90 (13) 93 (21) 87 (36) MRT (hours) 123 (14) 129 (19) 119 (31) L (ml/h/kg) 1.18 (28) 1.06 (29) 1.08 (39) Vss (dl/kg) 1.42 (24) 1.32 (20) 1.16 (14) Time to 1% Fctor IX 17 (29) 20 (22) 19 (37) ctivity (dys) * Time to 3% Fctor IX 12 (32) 14 (22) 13 (38) ctivity (dys) * Time to 5% Fctor IX 9 (32) 11 (26) 11 (38) ctivity (dys) * * = Estimted time to men Fctor IX ctivity mintined ove the pre-specified percent (%) IR = incrementl recovery recorded 30 minutes fter injection; U = re under the Fctor IX ctivity time curve; t 1/2 = hlf-life; MRT = men residence time; L = ody weight djusted clernce; Vss = ody weight djusted volume of distriution t stedy-stte. 13 NONLINIL TOXIOLOGY 13.1 rcinogenesis, Mutgenesis, Impirment of Fertility Nonclinicl studies evluting the crcinogenic potentil of IDELVION hve not een conducted. No mcroscopic or microscopic pthologies in reproductive orgns were oserved in nimls dosed every dy for 28 dys with 6.7 times the mximum recommended prophylctic clinicl dose of 75 IU/kg IDELVION. No niml studies regrding impirment of fertility following IDELVION dosing were conducted. 14 LINIL STUDIES The sfety nd efficcy of IDELVION were evluted in prospective, open-lel, multicenter clinicl study of 63 mle PTPs with hemophili ( 2% endogenous Fctor IX ctivity) who received t lest one infusion of IDELVION s prt of on-demnd tretment nd control of leeding episodes, periopertive mngement of mjor nd minor surgicl, dentl, or other invsive procedures, routine prophylxis once every 7-, 10- or 14-dy intervls, or phrmcokinetic evlution. Sujects were ged 12 to 61 yers; including 7 dolescent sujects ged 12 to 17. Sujects were treted for up to 27 months.

11 On-demnd Tretment nd ontrol of leeding Episodes totl of 358 leeding events were treted with IDELVION. mong them, 204 (57%) leeding events were spontneous, 140 (39%) events were trumtic nd 14 (4%) events were unknown. In ddition, totl of 267 (75%) episodes were joint leeding events. Overll tretment efficcy ws ssessed for ech leeding episode y the investigtor sed on 4-point scle of excellent, good, moderte, or poor/no response. The efficcy of IDELVION for the on-demnd tretment nd control of leeding episodes is summrized in Tle 7. Tle 7. Efficcy* of IDELVION for the On-demnd Tretment nd ontrol of leeding Episodes Numer of leeding Episodes Requiring Tretment (n = 358) Numer of injections to tret leeding episodes 1 injection, n (%) 335 (94) 2 injections, n (%) 18 (5) >2 injections, n (%) 5 (1.4) ssessment of Efficcy* Excellent or Good, n (%) 337 (94) Moderte, n (%) 9 (2.5) Poor/no response, n (%) 1 (0.3) *Excellent: Pin relief nd/or unequivocl improvement in ojective signs of leeding nd no dditionl infusion required in order to chieve hemostsis; Good: Definite pin relief nd/or improvement in signs of leeding t pproximtely 8 hours fter the first infusion, ut my require second infusion; Moderte: Prole or slight eneficil effect, nd requires more thn two infusions to chieve hemostsis; Poor/no response: No improvement t ll or condition worsens, dditionl hemosttic intervention is required. Responses evluted t pproximtely 24 hours fter tretment. Periopertive Mngement of leeding In three clinicl studies, 13 sujects received IDELVION for periopertive mngement of 15 surgicl procedures. Dose ws individulized sed on suject s PK nd clinicl response to tretment. The 15 surgicl procedures included doule mstectomy, four knee replcements, hemorrhoidectomy, rhinoplsty, nevus excision, n nkle rthroplsty, n endoscopic mucosl resection, four complicted dentl surgeries nd one uncomplicted dentl surgery. Two of the four dentl surgeries were performed in children <12 yers of ge. The efficcy nlysis of IDELVION in periopertive mngement included 12 surgeries in 10 PTPs etween 12 nd 61 yers of ge nd 3 surgicl procedures in 3 children <12 yers of ge undergoing mjor or minor surgicl procedure, including dentl surgeries. Hemostsis ws ssessed y the investigtor/surgeon t wound closure (intropertive ssessment), 72 hours fter surgery or t hospitl dischrge nd t the end of the surgicl sustudy using 4-point scle of: excellent, good, fir or none. Of the 15 surgeries included in the intropertive ssessment of hemosttic response, 12 surgeries were ssessed s excellent (n=11) or good (n=1), nd three minor surgeries were not rted. t hospitl dischrge or t the end of the surgicl sustudy, 14 surgicl procedures hd rting of excellent (n=13) or good (n=1), nd one surgery ws not rted. Routine Prophylxis Of the 63 sujects treted with IDELVION, twenty-three PTPs received IDELVION only for the tretment of leeding episodes during the first 6 months of the study. Nineteen of these PTPs switched to once weekly prophylxis with dditionl medin durtion of 10 months. sed on the nlysis of the 19 sujects treted with IDELVION for on-demnd therpy nd weekly prophylxis, the medin nnulized spontneous leeding rte (sr) during prophylxis tretment ws zero (rnge: 0 to 4.2), s compred to 15.4 (rnge: 2 to 39.5) during on-demnd tretment. sed on the Poisson model, prophylxis tretment with IDELVION resulted in 96% reduction in the nnulized spontneous leeding rte. The medin nnulized leeding rte (totl R spontneous nd trumtic) during prophylxis tretment ws 1.6; (rnge: 0 to 21.1), s compred to 19.2; (rnge: 2 to 46.1) during on-demnd tretment. sed on the Poisson model, prophylxis tretment with IDELVION resulted in n 89% reduction in the nnulized leeding rte. Tle 8. omprison of nnulized leeding Rtes for Sujects Treted for On-demnd Therpy nd Weekly Prophylxis leeding Episode Etiology Spontneous Men (SD) Medin IQR Rnge Totl Men (SD) Medin IQR Rnge On-demnd (n=19)* 14.6 (8.42) , , (9.19) , , 46.1 Weekly Prophylxis (n=19)* 0.7 (1.17) 0 0, 1.0 0, (4.81) 1.6 0, 4.1 0, 21.1 Percent Reduction with Prophylxis (n=19)* 96.0 (5.54) , , (17.76) , , 100 * sed on mtched pirs design Totl includes spontneous nd trumtic leeds IQR=interqurtile rnge, defined for 25 th percentile nd 75 th percentile; SD=stndrd devition; Sujects evlule for efficcy re sujects who received t lest one dose of on-demnd tretment, nd one dose of prophylxis tretment. Forty sujects received weekly routine prophylxis. Thirty-seven sujects completed 6 months of once weekly prophylxis. Of these, 21 sujects switched to 14-dy intervl with IU/kg of IDELVION with medin durtion of 12.7 months. Prior to switching, these 21 sujects were well controlled with IDELVION (did not require dose djustments or experience spontneous leeding 1 month prior to switch nd were mintined on prophylxis dose of 40 IU/kg every 7 dys). The medin srs for the 21 sujects treted with weekly nd 14-dy prophylxis were zero (rnge: 0 to 4.5) nd zero (rnge: 0 to 7.3), respectively (summrized in Tle 9). In ddition, the medin srs for the 7 sujects treted with weekly nd 10-dy prophylxis were zero (rnge: 0 to 0) nd zero (rnge: 0 to 0.9), respectively. Tle 9. omprison of nnulized Spontneous leeding Rte (sr) y Prophylxis Regimen for Sujects in the linicl Tril leeding Episode Etiology Spontneous Men (SD) Medin IQR Rnge Weekly Prophylxis (n=21) * 0.28 (1.01) 0 0, 0 0, dy Prophylxis (n=21) * 1.07 (2.1) 0 0, 1 0, 7.3 * sed on mtched pirs design IQR = interqurtile rnge; SD = Stndrd devition 15 REFERENES 1. Wilmot HV, Hogwood J, Gry E. Recominnt fctor IX: discrepncies etween onestge clotting nd chromogenic ssys. Hemophili 2014, Fritsm G, Demitzer FR, Rndhw, et l. Recommendtions for pproprite ctivted Prtil Thromoplstin Time Regent Selection nd Utiliztion. m J lin Pthol 2012;137: Sommer JM, uyue Y, rdn S, Peters RT, Jing H, Kmphus G, Gry E, Pierce GF. omprtive field study: impct of lortory ssy vriility on the ssessment of recominnt fctor IX Fc fusion protein (rfixfc) ctivity. Thromosis nd Hemostsis 2014, 112(5): HOW SUPPLIED/STORGE ND HNDLING How Supplied IDELVION is supplied s lyophilized powder in single-use vils contining the leled mount of Fctor IX ctivity, expressed in interntionl units (IU). IDELVION is pckged with 2.5 ml (for reconstitution of 250, 500 or 1000 IU vils) or 5 ml (for reconstitution of 2000 or 3500 IU vils) of Sterile Wter for Injection, USP, one Mix2Vil filter trnsfer set, nd one sterile lcohol sw. omponents re not mde with nturl ruer ltex. Nominl Strength Fill Size (Interntionl Units) olor Indictor Kit ND 250 Ornge lue Green Purple Yellow Storge nd Hndling Store IDELVION in its pckge to protect from light. Store the IDELVION pckge in the refrigertor or t room temperture 2-25 (36 to 77 F). Do not freeze. Do not use IDELVION or the Sterilized Wter for Injection diluent eyond the expirtion dte printed on the crton nd vil lels. 17 PTIENT OUNSELING INFORMTION dvise ptients to red the FD-pproved ptient leling (Ptient Informtion nd Instructions for Use). dvise ptients to report ny dverse rections or prolems following IDELVION dministrtion to their helthcre provider. Inform ptients of the erly signs nd symptoms of hypersensitivity or llergic rections (including hives, generlized urticri, chest tightness, wheezing, nd hypotension). Instruct ptients to discontinue use of IDELVION nd contct their helthcre provider nd/or seek immedite emergency cre if these symptoms occur [see Wrnings nd Precutions (5.1)]. dvise ptients to contct their helthcre provider or hemophili tretment center for further tretment nd/or ssessment if they experience lck of clinicl response to Fctor IX replcement therpy, s in some cses this my e mnifesttion of n inhiitor [see Wrnings nd Precutions (5.2)]. Mnufctured y: SL ehring GmH Mrurg, Germny for: SL ehring Recominnt Fcility G ern 22, Switzerlnd 3000 US License No Distriuted y: SL ehring LL Knkkee, IL US IDELVION is mnufctured under license y Novozymes iophrm /S, gsverd, Denmrk (US ptents pending). Mix2Vil is registered trdemrk of West Phrm. Services IL, Ltd., susidiry of West Phrmceuticl Services, Inc.