Drug Use Criteria: Complement Inhibitor and Enzyme/Protein Replacement Therapy

Transcription

1 Texas Vedor Drug Program Drug Use Criteria: Complemet Ihibitor ad /Protei Therapy Publicatio History 1. Developed December Revised April 2018; March 2018; March 2017; April 2015; March 2015; February Notes: Iformatio o idicatios for use or diagosis is assumed to be uavailable. All criteria may be applied retrospectively; prospective applicatio is idicated with a asterisk [*]. The iformatio cotaied is for the coveiece of the public. The Texas Health ad Huma Services Commissio is ot resposible for ay errors i trasmissio or ay errors or omissios i the documet. Medicatios listed i the tables ad o-fda approved idicatios icluded i these retrospective criteria are ot idicative of Vedor Drug Program formulary coverage. Prepared by: Drug Iformatio Service, UT Health Sa Atoio. The College of Pharmacy, the Uiversity of Texas at Austi. i

2 1 Complemet ihibitor ad ezyme/protei replacemet therapy is FDA-approved for coditios characterized by ezyme deficiecies such as adeosie deamiase (ADA) deficiecy i severe combied immuodeficiecy (SCID) patiets (pegademase bovie), atypical hemolytic uremic sydrome (eculizumab), cogeital sucrase isomaltase deficiecy (CSID) (sacrosidase), Fabry disease (agalsidase beta), Gaucher disease (imiglucerase, taliglucerase alfa, velaglucerase alfa), hereditary agioedema (HAE) (C1 esterase ihibitor, ecallatide, icatibat), hypophosphatasia (asfotase), lysosomal acid lipase (LAL) deficiecy (sebelipase alfa), mucopolysaccharidoses (MPS) [Maroteaux-Lamy sydrome (MPS VI) - galsulfase; Huter sydrome (MPS II) idursulfase; Hurler ad Hurler-Scheie forms of MPS 1 laroidase; Morquio A sydrome (MPS IVA) elosulfase; Sly sudrome (MPS VII vestroidase-alfa-vjbk)], myastheia gravis ad paroxysmal octural hemoglobiuria (eculizumab), Pompe disease (alglucosidase alfa) ad severe cogeital protei C deficiecy (protei C cocetrate). [1-34] 1.1 Adults Recommeded doses for complemet ihibitor as well as ezyme/protei replacemet therapy FDA-approved for use i adults are summarized i Table 1. Patiet profiles cotaiig doses exceedig maximum recommedatios will be reviewed. Table 1. Adult Complemet Ihibitor ad /Protei Therapy s[ 1-16,18-34] Type Treatmet Idicatio Stregth Recommeded Complemet Ihibitors Hereditary agioedema (HAE) treatmet (Beriert ) C1 esterase ihibitor, huma (Beriert ) 500 iteratioal uit (IU) for recostitutio 20 IU/kg by IV ijectio as a sigle dose 1

3 Treatmet Idicatio Stregth Recommeded Complemet Ihibitors HAE attacks, routie prevetio (Ciryze ) C1 esterase ihibitor, huma (Ciryze ) 500 uit for recostitutio 1000 U by IV ifusio every 3-4 days+ HAE attacks, routie prevetio (Haegarda ) C1 esterase ihibitor, huma (Haegarda ) 2000, 3000 IU s for recostitutio 60 IU/kg subcutaeousl y twice weekly (every 3 to 4 days) Complemet Ihibitors HAE treatmet C1 esterase ihibitor, recombiat (Rucoest ) 2100 IU/14 ml sigleuse for recostitutio < 84 kg: 50 uits/kg as sigle IV dose up to a maximum of 4200 uits per dose; may repeat x1 if attack symptoms persist i 24 hour period 84 kg: 4200 uits as a sigle IV dose; may repeat x1 if attack symptoms persist i 24 hour period Complemet Ihibitors HAE treatmet ecallatide (Kalbitor ) 10 mg/ml s x 3 30 mg subcutaeousl y as three separate 10 mg ijectios; may repeat x1 i 24 hour period if attack symptoms persist 2

4 Treatmet Idicatio Stregth Recommeded Complemet Ihibitors Atypical hemolytic uremic sydrome eculizumab (Soliris ) 300 mg sigle-dose 1200 mg as IV ifusio over 35 miutes Complemet Ihibitors Myestheia gravis, geeralized eculizumab (Soliris ) 1200 mg as IV ifusio over 35 miutes Complemet Ihibitors Paroxysmal octural hemoglobiuri a eculizumab (Soliris ) 900 mg as IV ifusio over 35 miutes Complemet Ihibitors HAE treatmet icatibat acetate (Firazyr ) 30 mg/3 ml syrige 30 mg subcutaeousl y as sigle dose; may repeat x2 with 6 hours betwee doses i 24 hour period if attack symptoms persist (maximum 3 doses per 24 hour period) Fabry disease agalsidase beta (Fabrazyme ) 5 mg, 35 mg s 1 mg/kg by itraveous (IV) ifusio every 2 Pompe disease alglucosidase alfa (Lumizyme ) 50 mg 20 mg/kg as a IV ifusio every 2 3

5 Treatmet Idicatio Stregth Recommeded Hypophosphat asia (periatal/ifa tile- or juveileoset) asfotase alfa (Stresiq ) 18 mg/0.45 ml, 28 mg/0.7 m L, 40 mg/ml, or 80 mg/0.8 m L s Hypophosphat asia (periatal/ifa tile-oset#): birth to ay age: 9 mg/kg weekly as subcutaeous ijectio as 3 mg/kg three times weekly Hypophosphat asia (juveileoset^): 6 moths ad older: 6 mg/kg weekly as subcutaeous ijectio, give either as 2 mg/kg three times weekly or 1 mg/kg six times weekly Mucopolysaccharidosis (MPS) IVA (Morquio A sydrome) elosulfase (Vimizim ) 5 mg/5 ml 2 mg/kg by IV ifusio over a miimum of 3.5 to 4.5 hours oce weekly MPS VI (Maroteaux- Lamy sydrome) galsulfase (Naglazyme ) 5 mg/5 ml preservativefree s 1 mg/kg by IV ifusio oce weekly Huter sydrome idursulfase (Elaprase ) 6 mg/3 ml 0.5 mg/kg as IV ifusio oce weekly Gaucher disease, type 1 (oeuropat hic) imiglucerase (Cerezyme ) 200 mg, 400 mg s for recostitutio 60 U/kg by IV ifusio over 1-2 hours every 2 4

6 Treatmet Idicatio Stregth Recommeded MPS 1 (Hurler, Hurler-Scheie forms; Scheie form with moderate to severe symptoms) laroidase (Aldurazyme ) 2.9 mg/5 ml s 0.58 mg/kg by IV ifusio over 3-4 hours oce weekly Cogeital sucraseisomaltase deficiecy (CSID) sacrosidase (Sucraid ) 8500 iteratioal uits/ml as 118 ml oral solutio bottles 15 kg: 8500 uits orally mixed i 2-4 ouces of water or milk with each meal or sack > 15 kg: 17,000 uits orally mixed i 2-4 ouces of water or milk with each meal or sack Lysosomal acid lipase (LAL) deficiecy sebelipase alfa (Kauma ) 20 mg/10 ml sigleuse 1 mg/kg by IV ifusio oce Gaucher disease, type 1 taliglucerase alfa (Elelyso ) 200 uit s for recostitutio Treatmetaïve: 60 U/kg by IV ifusio oce every 2 Previously treated with imiglucerase: use same uit/kg dosage for taliglucerase that was prescribed for imiglucerase ad admiister 5

7 Treatmet Idicatio Stregth Recommeded Gaucher disease, type 1 velagluceras e alfa (Vpriv ) 400 uit s for recostitutio 60 U/kg as a IV ifusio every 2 MPS VII (Sly sydrome), excludig cetral ervous system symptoms vestroidase alfa-vjbk (Mepsevii ) 10 mg/5 ml 4 mg/kg as IV ifusio every two Protei Protei C deficiecy (acute episode*) protei C cocetrate (Ceproti ) 500 IU, 1000 IU for recostitutio IU/kg iitial dose by IV ifusio, followed by IU/kg every 6 hours for 3 doses by IV ifusio Protei Protei C deficiecy (short-term prophylaxis/ maiteace dose*) protei C cocetrate (Ceproti ) IU/kg every 6 to 12 hours by IV ifusio Protei Protei C deficiecy (log-term prophylaxis*) protei C cocetrate (Ceproti ) IU/kg every 12 hours by IV ifusio + = I patiets ot respodig adequately, doses up to 2500 uits (ot exceedig 100 u/kg) every 3 or 4 days may be utilized based o idividual patiet respose # = Hypophosphatasia, periatal/ifatile oset = symptoms develop at < 6 moths of age ^ = Hypophosphatasia, juveile-oset = symptoms occur betwee 6 moths ad 18 years of age * = protei C cocetrate ifusio rate: 2 ml/mi 6

8 1.2 Pediatrics Pegademase bovie is FDA-approved for use i eoates, ifats ad childre with ADA deficiecy due to severe combied immuodeficiecy who have failed or are ot cadidates for boe marrow trasplatatio; pegademase bovie safety ad efficacy have ot bee established i adults. [17, 25-27] C1 esterase ihibitor safety ad efficacy have ot bee determied i pediatric patiets youger tha 13 years of age. [1-4, 25-27] recommeded dosages for complemet ihibitor ad protei/ezyme replacemet therapies FDA-approved for use i pediatric patiets are summarized i Table 2. s exceedig these recommedatios will be reviewed. Table 2. Pediatric Complemet Ihibitor ad /Protei 1-6, 8-34 Therapy s Type Treatmet Idicatio Stregth Recommeded Complemet Ihibitors Hereditary agioedema (HAE) treatmet (Beriert ) C1 esterase ihibitor, huma (Beriert ) 500 uit for recostitutio 5 to 17 years: 20 IU/kg by IV ijectio as a sigle dose Complemet Ihibitors HAE attacks, routie prevetio (Ciryze ) C1 esterase ihibitor, huma (Ciryze ) 500 uit for recostitutio 13 to 17 years: 1000 U by IV ifusio every 3-4 days+ Complemet Ihibitors HAE attacks, routie prevetio (Haegarda ) C1 esterase ihibitor, huma (Haegarda ) 2000, 3000 IU s for recostitutio 12 to 17 years: 60 IU/kg subcutaeously twice weekly (every 3 to 4 days) 7

9 Treatmet Idicatio Stregth Recommeded Complemet Ihibitors HAE treatmet C1 esterase ihibitor, recombia t (Rucoest ) 2100 IU/14 ml sigleuse for recostitutio 13 to 17 years (< 84 kg): 50 uits/kg as sigle IV dose up to a maximum of 4200 uits per dose; may repeat x1 if attack symptoms persist i 24 hour period 13 to 17 years (> 84 kg): 4200 uits as a sigle IV dose; may repeat x1 if attack symptoms persist i 24 hour period Complemet Ihibitors HAE treatmet ecallatide (Kalbitor ) 10 mg/ml s x 3 12 to 17 years: 30 mg subcutaeously as three separate 10 mg ijectios; may repeat x1 i 24 hour period if attack symptoms persist 8

10 Treatmet Idicatio Stregth Recommeded Complemet Ihibitors Atypical hemolytic uremic sydrome eculizumab (Soliris ) 300 mg sigle-dose > 2 moths (5-9 kg): 300 mg by IV ifusio over 1-4 hours every 3 childre, adolescets kg: 300 mg by IV ifusio over 1-4 hours childre, adolescets kg: 600 mg by IV ifusio over 1-4 hours childre, adolescets kg: 900 mg by IV ifusio over 1-4 hours childre, adolescets 40 kg: 1200 mg by IV ifusio over 1-4 hours every two Fabry disease agalsidase beta (Fabrazyme ) 5 mg, 35 mg s 8-17 years: 1 mg/kg by itraveous (IV) ifusio every 2 Pompe disease alglucosidas e alfa (Lumizyme ) 50 mg 1 moth of age ad older: 20 mg/kg as a IV ifusio every 2 9

11 Treatmet Idicatio Stregth Recommeded Hypophospha tasia (periatal/if atile- or juveileoset) asfotase alfa (Stresiq ) 18 mg/0.45 ml, 28 mg/0.7 m L, 40 mg/ml, or 80 mg/0.8 m L s Hypophosphata sia (periatal/ifa tile-oset#): birth to ay age: 9 mg/kg weekly as subcutaeous ijectio as 3 mg/kg three times weekly Hypophosphata sia (juveileoset^): 6 moths ad older: 6 mg/kg weekly as subcutaeous ijectio, give either as 2 mg/kg three times weekly or 1 mg/kg six times weekly Late ifatile euroal ceroid lipofusciosis type 2 (CLN2) disease cerlipoase alfa (Brieura ) 150 mg/5 ml as 2 sigleuse s copackaged with itravetricul ar electrolytes 3 years ad older: 300 mg every other week by itravetricular ifusio, followed by itravetricular electrolytes Mucopolysaccharidosis (MPS) IVA (Morquio A sydrome) elosulfase (Vimizim ) 5 mg/5 ml 5 years ad older: 2 mg/kg by IV ifusio over a miimum of hours oce weekly MPS VI (Maroteaux- Lamy sydrome) galsulfase (Naglazyme ) 5 mg/5 ml preservativefree s 5 years ad older: 1 mg/kg by IV ifusio oce weekly 10

12 Treatmet Idicatio Stregth Recommeded Huter sydrome idursulfase (Elaprase ) 6 mg/3 ml 5 to 17 years: 0.5 mg/kg as IV ifusio oce weekly Gaucher disease, type 1 (oeuropat hic) imigluceras e (Cerezyme ) 200 mg, 400 mg s for recostitutio 2 to 16 years: 60 U/kg by IV ifusio over 1-2 hours every 2 MPS 1 (Hurler, Hurler-Scheie forms; Scheie form with moderate to severe symptoms) laroidase (Aldurazym e ) 2.9 mg/5 ml s 6 moths of age ad older: 0.58 mg/kg by IV ifusio over 3-4 hours oce weekly Severe combied immuedeficiecy associated with adeosie deamiase deficiecy pegademas e (Adage ) 375 uits/1.5 ml birth to 12 years: 30 uits/kg as a sigle dose per week Cogeital sucraseisomaltase deficiecy (CSID) sacrosidase (Sucraid ) 8500 iteratioal uits/ml as 118 ml oral solutio bottles 5 moths to 17 years: 15 kg: 8500 uits orally mixed i 2-4 ouces of water, milk, or ifat formula with each meal or sack > 15 kg: 17,000 uits orally mixed i 2-4 ouces of water or milk with each meal or sack 11

13 Treatmet Idicatio Stregth Recommeded Lysosomal acid lipase (LAL) deficiecy sebelipase alfa (Kauma ) 20 mg/10 ml sigleuse 1 moth of age to 17 years: 1 mg/kg by IV ifusio oce Rapidly progressive LAL deficiecy (developig i first 6 moths of life) sebelipase alfa (Kauma ) 1 moth of age to 17 years: 3 mg/kg by IV ifusio oce weekly Gaucher disease, type 1 taligluceras e alfa (Elelyso ) 200 uit s for recostitutio Treatmetaïve (4 years ad older): 60 U/kg by IV ifusio oce every 2 Previously treated with imiglucerase (4 years ad older): use same uit/kg dosage for taliglucerase that was prescribed for imiglucerase ad admiister 12

14 Treatmet Idicatio Stregth Recommeded Gaucher disease, type 1 velaglucera se alfa (Vpriv ) 400 uit s for recostitutio Treatmetaïve (4 years ad older): 60 U/kg by IV ifusio oce every 2 Previously treated with imiglucerase (4 years ad older): use same uit/kg dosage for taliglucerase that was prescribed for imiglucerase ad admiister MPS VII (Sly sydrome), excludig cetral ervous system symptoms vestroidas e alfa-vjbk (Mepsevii ) 10 mg/5 ml birth to 17 years: 4 mg/kg as IV ifusio Protei Protei C deficiecy (acute episode*) protei C cocetrate (Ceproti ) 500 IU, 1000 IU for recostitutio birth to 17 years: IU/kg iitial dose by IV ifusio, followed by IU/kg every 6 hours for 3 doses by IV ifusio* Protei Protei C deficiecy (short-term prophylaxis/ maiteace dose*) protei C cocetrate (Ceproti ) birth to 17 years: IU/kg every 6 to 12 hours by IV ifusio 13

15 Treatmet Idicatio Stregth Recommeded Protei Protei C deficiecy (log-term prophylaxis*) protei C cocetrate (Ceproti ) birth to 17 years: IU/kg every 12 hours by IV ifusio + = I patiets ot respodig adequately, doses up to 2500 uits (ot exceedig 100 u/kg) every 3 or 4 days may be utilized based o idividual patiet respose # = Hypophosphatasia, periatal/ifatile oset = symptoms develop at < 6 moths of age ^ = Hypophosphatasia, juveile-oset = symptoms occur betwee 6 moths ad 18 years of age * = protei C cocetrate ifusio rate: 2 ml/mi, except i childre < 10 kg, where ifusio rate should ot exceed 0.2 ml/kg/mi Although ot FDA-approved, some ivestigators have studied agalsidase use i childre youger tha 8 years of age to reduce or prevet complicatios associated with Fabry disease (e.g., kidey complicatios, cardiovascular disease, cerebrovascular dysfuctio). Studies have icluded patiets ragig i age from 2.5 to 8 years of age for boys ad 4.4 to 8 years of age for girls. Results, based o small patiet umbers, have show improvemets i disease maifestatios, pai ad quality of life without sigificat adverse effects i youger childre. Further, [35, 36] log-term trials are ecessary to cofirm these results. Although ot FDA-approved, alglucosidase alfa has bee evaluated for early use to treat Pompe disease. I a small study, ivestigators foud that alglucosidase therapy iitiated early after diagosis i eoates less tha 1 moth of age ca improve cliical outcomes eve before oset of cliical symptoms i ifats with Pompe disease. Further, log-term trials are eeded to corroborate these fidigs. [37] 2 Duratio of Therapy [1-34] There is o basis for limitig the duratio of complemet ihibitor ad ezyme/protei replacemet therapy as ezyme deficiecies represet chroic disorders ad require sustaied treatmet.duplicative Therapy 14

16 FDA-approved ezyme replacemet therapies are idicated for specific ezyme deficiecies. Patiets with multiple ezyme deficiecies may be prescribed multiple ezyme replacemet therapies cocurretly. Adjuctive admiistratio of ezyme replacemet therapies without multiple ezyme deficiecy diagoses is ot cliically reasoable ad will be evaluated. 3 Refereces 1. C1 esterase ihibitor, huma (Beriert ) package isert. CSL Behrig, September C1 esterase ihibitor, huma itraveous ijectio (Ciryze ) package isert. Shire ViroPharma, Ic., December C1 esterase ihibitor subcutaeous ijectio (Haegarda ) package isert. CSL Behrig, October C1 esterase ihibitor, recombiat (Rucoest ) package isert. Salix Pharmaceuticals, Ic., February Ecallatide subcutaeous ijectio (Kalbitor ) package isert. Shire, March Eculizumab itraveous ijectio (Soliris ) package isert. Alexio Pharmaceuticals, Ic., October Icatibat subcutaeous ijectio (Firazyr ) package isert. Shire Orpha Therapies LLC, December Agalsidase beta ijectio (Fabrazyme ) package isert. Gezyme Corporatio, July Alglucosidase alfa ijectio (Lumizyme ) package isert. Gezyme Corporatio, August Asfotase alfa ijectio (Stresiq ) package isert. Alexio Pharmaceuticals, Ic., October Cerlipoase alfa ijectio (Brieura ) package isert. BioMari Pharmaceutical Ic., April Elosulfase ijectio (Vimizim ) package isert. BioMari Pharmaceutical Ic., February Galsulfase ijectio (Naglazyme ) package isert. BioMari Pharmaceutical Ic., March Idursulfase ijectio (Elaprase ) package isert. Shire Huma Geetic Therapies, Ic., Jue

17 15.Imiglucerase ijectio (Cerezyme ) package isert. Gezyme Corporatio, May Laroidase itraveous ifusio (Aldurazyme ) package isert. BioMari Pharmaceutical Ic., April Pegademase bovie ijectio (Adage ) package isert. Leadiat Scieces, November Sacrosidase oral solutio (Sucraid ) package isert. QOL Medical, LLC, March Sebelipase ijectio (Kauma ) package isert. Alexio Pharmaceuticals, Ic., December Taliglucerase alfa ijectio (Elelyso ) package isert. Pfizer, December Velaglucerase alfa ijectio (Vpriv ) package isert. Shire Huma Geetic Therapies, Ic., April Vestroidase alfa-vjbk ijectio (Mepsevii ) package isert. Ultrageyx Pharmaceutical Ic., November Protei C cocetrate (Ceproti ) package isert. Baxalta US Ic., September DRUGDEX System (electroic versio). Truve Health Aalytics, Greewood Village, Colorado, USA. Available at: Accessed March 14, Cliical Pharmacology [database olie]. Tampa, FL: Gold Stadard, Ic; Available at: Accessed March 14, Facts ad Comparisos easwers [database olie]. Hudso, Ohio: Wolters Kluwer Cliical Drug Iformatio, Ic.; Available at: fcofactsadcomparisos-com.ezproxy.lib.utexas.edu/. Accessed March 14, AHFS Drug Iformatio Jackso, WY: Teto Data Systems, Versio , Stat!Ref Electroic Medical Library. Available at: olie.statref.com.libproxy.uthscsa.edu/. Accessed March 14, Wy R. Mucopolysaccharidoses: complicatios ad maagemet. Post TW, ed. UpToDate. Waltham, MA: UpToDate Ic. (Accessed March 14, 2018). 29.Merritt JL. Lysosomal acid alpha-glucosidase deficiecy (Pompe disease, glycoge storage disease II, acid maltase deficiecy). Post TW, ed. UpToDate. Waltham, MA: UpToDate, Ic. (Accessed o March 14, 2018). 16

18 30.Muezer J, Beck M, Eg CM, et al. Multidiscipliary maagemet of Huter sydrome. Pediatrics. 2009; 124(6):e1228-e Valayaopoulos V, Wijburg FA. Therapy for the mucopolysaccharidoses. Rheumatology. 2011; 50:v49-v Zarate YA, Hopki RJ. Fabry s disease. Lacet. 2008; 372: va der Ploeg AT, Reuser AJJ. Pompe s disease. Lacet. 2008; 372: Che M, Wag J. Gaucher disease. Review of the literature. Arch Pathol Lab Med. 2008; 132: Ramaswami U, Wedt S, Pitos-Morell G, et al. replacemet therapy with agalsidase alfa i childre with Fabry disease. Acta Paediatrica. 2007; 96(1): Ramaswami U, Parii R, Kampma C, Beck M. Safety of agalsidase alfa i patiets with Fabry disease uder 7 years. Acta Paediatrica. 2011; 100(4): Chie YH, Lee NC, Thurberg BL, et al. Pompe disease i ifats: improvig the progosis by ewbor screeig ad early treatmet. Pediatrics. 2009; 124(6):e