Download Starting treatment with vpriv

Starting treatment with vpriv
Steps to accessing personalized therapy
If you have a patient who is ready to begin treatment with VPRIV, please follow these steps:
STEP 1: Consider discussing OnePath with patient
Talk to your patient and/or the family about OnePath, an optional product support services program.
The OnePath patient brochure explains how OnePath may offer assistance to those starting treatment
with VPRIV.
Step 2: Complete forms
Call a OnePath Case Manager at 1-866-888-0660 or visit www.vpriv.com to access the necessary forms.
Physicians must complete and sign the Start Form (VPRIV Prescription and OnePath Start Form) as well as
the Statement of Medical Necessity form.
• If your patient (or his/her guardian) is interested in receiving OnePath product support services,
he/she needs to sign the Start Form as well (patients do not need to sign the Start Form to
receive therapy with VPRIV; they need to sign it only if they would like access to OnePath product
support services)
Step 3: Submit forms
Fax the signed Start Form, Statement of Medical Necessity form, and a copy of your patient’s insurance
card(s) to OnePath at 1-888-990-0008.
Step 4: Your patient will be contacted by a Case Manager
Once the signed Start Form showing your patient has elected to access OnePath product support
services is received, a OnePath Case Manager will contact your patient. The Case Manager will
review all the details of the product support services, as outlined below, and set appropriate expectations
for starting therapy.
If you have any questions, call OnePath toll-free at 1-866-888-0660 from 8:30 AM to 8:00 PM Eastern Time,
Monday through Friday.
How onepath works
Your Case Manager receives the completed Start Form signed by you and your patient; then:
Welcomes patient and sets appropriate expectations
for therapy.
Assists with locating an infusion site, if necessary.
Verifies insurance coverage and refers to additional sources
of financial/reimbursement assistance, if necessary.
Helps to ensure treatment supply by facilitating delivery of
VPRIV to the site of care.
Helps to ensure that appointments are made and kept,
and will follow up, if necessary.
Provides ongoing product support for your patient and
staff, answering disease- or treatment-related questions
throughout the process, as needed.
Please see Important Safety Information on reverse.
Please see accompanying full Prescribing Information.
It all comes together.
GUIDANCE ON billing information for vpriv
ICD-9-CM:
272.7 Lipidosis (Gaucher disease)
NDC:
54092-701-04 400-unit vial
HCPCS:
J3385 (Injection, velaglucerase alfa, 100 units)
CPT-4:
96365 Intravenous infusion therapy, prophylaxis, or
diagnosis (specify substance or drug); initial, up to 1 hour
96366 Each additional hour (list separately in addition to
primary procedure code, 96365)
Revenue:
260 General IV therapy service
261 Infusion pump
258 IV solutions
636 Drugs and biologicals requiring a HCPCS code
Providers are responsible
for selecting the appropriate
codes. This table serves as
a general guide for possible
coding alternatives and
should not be a substitute for
a healthcare professional’s
own judgment.
Indication
VPRIV is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy (ERT)
for pediatric and adult patients with type 1 Gaucher disease.
Important Safety Information
• The most serious adverse reactions in patients treated with VPRIV were hypersensitivity reactions. Appropriate medical
support should be available when VPRIV is administered. If a severe reaction occurs, medical standards for emergency
treatment are to be followed.
• Treatment with VPRIV should be used with caution in patients who have exhibited symptoms of hypersensitivity to the active
ingredient, drug product excipients, or to other enzyme replacement therapies.
• Infusion-related reactions were the most commonly observed adverse reactions in patients treated with VPRIV in clinical
studies. The most commonly observed symptoms of infusion-related reactions were: headache, dizziness, hypotension,
hypertension, nausea, asthenia/fatigue, and pyrexia. Generally the infusion-related reactions were mild and, in treatmentnaïve patients, onset occurred mostly during the first 6 months of treatment and tended to occur less frequently with time.
• Management of infusion-related reactions should be based on severity of the reaction, such as slowing the infusion rate,
treatment with medications such as antihistamines, antipyretics and/or corticosteroids, and/or stopping and resuming
treatment with increased infusion time.
• Other commonly observed adverse reactions in ≥10% of ERT-naïve or imiglucerase-switched patients were: headache,
dizziness, abdominal pain, nausea, back pain, joint pain, upper respiratory tract infection, activated PTT prolonged, infusionrelated reactions, pyrexia, and asthenia/fatigue.
• All adult adverse reactions to VPRIV are considered relevant to pediatric patients (ages 4 to 17 years). Adverse reactions more
common in pediatric patients (>10% difference) included upper respiratory tract infection, rash, aPTT prolonged, and pyrexia.
The safety of VPRIV has not been established in pediatric patients younger than 4 years of age.
• As with all therapeutic proteins, there is a potential for immunogenicity. In clinical studies, 1 of 54 treatment-naïve patients
treated with VPRIV (who received a 45 Units/kg dose) developed IgG class antibodies (neutralizing in an in vitro assay). It is
unknown if the presence of IgG antibodies to VPRIV is associated with a higher risk of infusion reactions. Patients with an
immune response to other enzyme replacement therapies who are switching to VPRIV should continue to be monitored
for antibodies.
• For more information, please see the full prescribing information or call Shire at 1-866-888-0660. More information about
VPRIV can also be obtained by going to www.vpriv.com. To report suspected adverse events contact Shire Human Genetic
Therapies, at the OnePathSM phone number 1-866-888-0660 or [email protected], or FDA at 1-800-FDA-1088 or
www.fda.gov/medwatch.
Please see accompanying full Prescribing Information.
www.vpriv.com
OnePath is service mark and VPRIV is a registered trademark of
Shire Human Genetic Therapies, Inc.
Shire Human Genetic Therapies, Inc. 700 Main St., Cambridge, MA 02139
©2010 Shire Human Genetic Therapies, Inc. US/VEL-00220-Dec10
It all comes together.
40-0510 Rev.
TM
(velaglucerase alfa
for injection)
TM
1234567890
40-0510 Rev.
(velaglucerase alfa
for injection)
HIGHLIGHTS OF PRESCRIBING INFORMATION
These highlights do not include all the information needed to use VPRIV safely and effectively. See full
prescribing information for VPRIV.
FULL PRESCRIBING INFORMATION: CONTENTS*
1
INDICATIONS AND USAGE
VPRIV— (velaglucerase alfa for injection)
Initial U.S. Approval: 2010
2
DOSAGE AND ADMINISTRATION
3FDPNNFOEFE%PTF
1SFQBSBUJPOBOE"ENJOJTUSBUJPO*OTUSVDUJPOT
--------------------------------INDICATIONS AND USAGE------------------------------VPRIV(velaglucerase alfa for injection) is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term
enzyme replacement therapy (ERT) for pediatric and adult patients with type 1 Gaucher disease (1).
3
DOSAGE FORMS AND STRENGTHS
4
CONTRAINDICATIONS
----------------------------DOSAGE AND ADMINISTRATION----------------------------
5
WARNINGS AND PRECAUTIONS
)ZQFSTFOTJUJWJUZ3FBDUJPOT
*OGVTJPOSFMBUFE3FBDUJPOT
r6OJUTLHBENJOJTUFSFEFWFSZPUIFSXFFLBTBNJOVUFJOUSBWFOPVTJOGVTJPO
r1BUJFOUTDVSSFOUMZCFJOHUSFBUFEXJUIJNJHMVDFSBTFGPS(BVDIFSEJTFBTFDBOCFTXJUDIFEUP713*71BUJFOUTQSFWJPVTMZUSFBUFE
on a stable dose of imiglucerase are recommended to begin treatment with VPRIV at that same dose when they switch
GSPNJNJHMVDFSBTFUP713*7
r1IZTJDJBOT DBO NBLF EPTBHF BEKVTUNFOUT CBTFE PO BDIJFWFNFOU BOE NBJOUFOBODF PG FBDI QBUJFOUT UIFSBQFVUJD HPBMT
$MJOJDBMUSJBMTIBWFFWBMVBUFEEPTFTSBOHJOHGSPN6OJUTLHUP6OJUTLHFWFSZPUIFSXFFL
------------------------DOSAGE FORMS AND STRENGTHS---------------------------r-ZPQIJMJ[FEQPXEFSUPCFSFDPOTUJUVUFEBOEEJMVUFEGPSJOGVTJPO
r"WBJMBCMFJO6OJUTBOE6OJUTTJOHMFVTFWJBMT
6
ADVERSE REACTIONS
$MJOJDBM4UVEJFT&YQFSJFODF
7
DRUG INTERACTIONS
8
USE IN SPECIFIC POPULATIONS
1SFHOBODZm$BUFHPSZ#
/VSTJOH.PUIFST
1FEJBUSJD6TF
(FSJBUSJD6TF
10
OVERDOSAGE
r/POF
11
DESCRIPTION
-----------------------------WARNINGS AND PRECAUTIONS--------------------------r)ZQFSTFOTJUJWJUZSFBDUJPOT5SFBUNFOUXJUI713*7TIPVMECFDBSFGVMMZSFFWBMVBUFEJOUIFQSFTFODFPGTJHOJGJDBOUFWJEFODFPG
hypersensitivity to the product (5.1).
r*OGVTJPOSFMBUFESFBDUJPOT
12
CLINICAL PHARMACOLOGY
.FDIBOJTNPG"DUJPO
1IBSNBDPLJOFUJDT
----------------------------------CONTRAINDICATIONS---------------------------------
---------------------------------ADVERSE REACTIONS---------------------------------r5IFNPTUDPNNPOBEWFSTFSFBDUJPOTEVSJOHDMJOJDBMTUVEJFTXFSFJOGVTJPOSFMBUFESFBDUJPOT
r0UIFSDPNNPOMZPCTFSWFEBEWFSTFSFBDUJPOTJOÿPGQBUJFOUTXFSFIFBEBDIFEJ[[JOFTTBCEPNJOBMQBJOOBVTFBCBDL
QBJOKPJOUQBJOVQQFSSFTQJSBUPSZUSBDUJOGFDUJPOBDUJWBUFE155QSPMPOHFEGBUJHVFBTUIFOJBBOEQZSFYJB
SM
To report SUSPECTED ADVERSE REACTIONS, contact Shire Human Genetic Therapies, Inc. at the OnePath
phone # 1-866-888-0660 or [email protected], or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch
13
NONCLINICAL TOXICOLOGY
$BSDJOPHFOFTJT.VUBHFOFTJT*NQBJSNFOUPG'FSUJMJUZ
14
CLINICAL STUDIES
14.1 Studies of VPRIV as Initial Therapy
4UVEZJO1BUJFOUT4XJUDIJOHGSPN*NJHMVDFSBTF5SFBUNFOUUP713*7
15
REFERENCES
16
HOW SUPPLIED/STORAGE AND HANDLING
4UPSBHF
17
PATIENT COUNSELING INFORMATION
See Section 17 for PATIENT COUNSELING INFORMATION.
3FWJTFE<>
*Sections or subsections omitted from the full prescribing information are not listed.
VPRIV™
5IFNPTUDPNNPOMZSFQPSUFEBEWFSTFSFBDUJPOTPDDVSSJOHJOÿPGQBUJFOUT
UIBUXFSFDPOTJEFSFESFMBUFEUP713*7BSFTIPXO
JO5BCMF5IFNPTUDPNNPOBEWFSTFSFBDUJPOTXFSFJOGVTJPOSFMBUFESFBDUJPOT
(velaglucerase alfa for injection)
FULL PRESCRIBING INFORMATION
1 INDICATIONS AND USAGE
VPRIV (velaglucerase alfa for injection) is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme
replacement therapy (ERT) for pediatric and adult patients with type 1 Gaucher disease.
2 DOSAGE AND ADMINISTRATION
2.1 Recommended Dose
5IFSFDPNNFOEFEEPTFJT6OJUTLHBENJOJTUFSFEFWFSZPUIFSXFFLBTBNJOVUFJOUSBWFOPVTJOGVTJPO
Patients currently being treated with imiglucerase for type 1 Gaucher disease may be switched to VPRIV. Patients previously
treated on a stable dose of imiglucerase are recommended to begin treatment with VPRIV at that same dose when they switch
from imiglucerase to VPRIV.
%PTBHFBEKVTUNFOUTDBOCFNBEFCBTFEPOBDIJFWFNFOUBOENBJOUFOBODFPGFBDIQBUJFOUTUIFSBQFVUJDHPBMT$MJOJDBMTUVEJFTIBWF
FWBMVBUFEEPTFTSBOHJOHGSPN6OJUTLHUP6OJUTLHFWFSZPUIFSXFFL
VPRIV should be administered under the supervision of a healthcare professional.
2.2 Preparation and Administration Instructions
Use aseptic technique
713*7JTBMZPQIJMJ[FEQPXEFSXIJDISFRVJSFTSFDPOTUJUVUJPOBOEEJMVUJPOBOEJTJOUFOEFEGPSJOUSBWFOPVTJOGVTJPOPOMZ713*7
DPOUBJOTOPQSFTFSWBUJWFTBOEWJBMTBSFTJOHMFVTFPOMZ%JTDBSEBOZVOVTFETPMVUJPO713*7TIPVMECFQSFQBSFEBTGPMMPXT
#FDBVTFDMJOJDBMUSJBMTBSFDPOEVDUFEVOEFSXJEFMZWBSZJOHDPOEJUJPOTBEWFSTFSFBDUJPOSBUFTPCTFSWFEJOUIFDMJOJDBMUSJBMTPGBESVH
cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
Table 2: Adverse Reactions Observed in ≥10% of Patients with Type 1 Gaucher Disease Treated with VPRIV
System Organ Class
Preferred Term
Naïve to ERT
N = 54
Switched from imiglucerase
to VPRIV
N = 40
Number of Patients (%)
Nervous system disorders
)FBEBDIF
Dizziness
4 (10)
Gastrointestinal disorders
"CEPNJOBMQBJO
%FUFSNJOFUIFOVNCFSPGWJBMTUPCFSFDPOTUJUVUFECBTFEPOUIFJOEJWJEVBMQBUJFOUTXFJHIUBOEUIFQSFTDSJCFEEPTF'PMMPXUIF
instructions in Table 1 for reconstitution.
/BVTFB
Table 1: Reconstitution Instructions
Musculoskeletal and connective tissue disorders
#BDLQBJO
200 Units/vial
400 Units/vial
+PJOUQBJOLOFF
7PMVNFPG4UFSJMF8BUFSGPS*OKFDUJPO641
for reconstitution
N-
N-
Infections and infestations
Concentration after reconstitution
6OJUTN-
6OJUTN-
Infusion-related reaction*
1ZSFYJB
Withdrawal volume
N-
N-
6QQFSSFTQJSBUPSZUSBDUJOGFDUJPO
Investigations
"DUJWBUFEQBSUJBMUISPNCPQMBTUJOUJNFQSPMPOHFE
6QPOSFDPOTUJUVUJPONJYWJBMTHFOUMZ%0/054)",&1SJPSUPGVSUIFSEJMVUJPOWJTVBMMZJOTQFDUUIFTPMVUJPOJOUIFWJBMTUIFTPMVUJPO
TIPVME CF DMFBS UP TMJHIUMZ PQBMFTDFOU BOE DPMPSMFTT EP OPU VTF JG UIF TPMVUJPO JT EJTDPMPSFE PS JG GPSFJHO QBSUJDVMBUF NBUUFS JT
QSFTFOU8JUIESBXUIFDBMDVMBUFEWPMVNFPGESVHGSPNUIFBQQSPQSJBUFOVNCFSPGWJBMTBOEEJMVUFUIFUPUBMWPMVNFSFRVJSFEJO
N-PGTPEJVNDIMPSJEFTPMVUJPOTVJUBCMFGPS*7BENJOJTUSBUJPO.JYHFOUMZ%0/054)",&
713*7TIPVMECFBENJOJTUFSFEPWFSNJOVUFT713*7TIPVMEOPUCFJOGVTFEXJUIPUIFSQSPEVDUTJOUIFTBNFJOGVTJPOUVCJOHBT
the compatibility in solution with other products has not been evaluated. The diluted solution should be filtered through an in-line
MPXQSPUFJOCJOEJOHíNGJMUFSEVSJOHBENJOJTUSBUJPO
"T713*7DPOUBJOTOPQSFTFSWBUJWFTPODFSFDPOTUJUVUFEUIFQSPEVDUTIPVMECFVTFEJNNFEJBUFMZ*GJNNFEJBUFVTFJTOPUQPTTJCMF
UIFSFDPOTUJUVUFEPSEJMVUFEQSPEVDUNBZCFTUPSFEGPSVQUPIPVSTBUUP$UP'
%POPUGSFF[F1SPUFDUGSPNMJHIU
5IFJOGVTJPOTIPVMECFDPNQMFUFEXJUIJOIPVSTPGSFDPOTUJUVUJPOPGWJBMT
3 DOSAGE FORMS AND STRENGTHS
713*7 JT B TUFSJMF XIJUF UP PGGXIJUF MZPQIJMJ[FE QPXEFS GPS SFDPOTUJUVUJPO XJUI 4UFSJMF 8BUFS GPS *OKFDUJPO 641 UP ZJFME B GJOBM
DPODFOUSBUJPOPG6OJUTN-
713*7JTBWBJMBCMFBT6OJUTBOE6OJUTTJOHMFVTFWJBMT
4 CONTRAINDICATIONS
/POF
5 WARNINGS AND PRECAUTIONS
5.1 Hypersensitivity Reactions
)ZQFSTFOTJUJWJUZ SFBDUJPOT IBWF CFFO SFQPSUFE JO QBUJFOUT JO DMJOJDBM TUVEJFT XJUI 713*7 <see Adverse Reactions (6.1)> "T XJUI
BOZJOUSBWFOPVTQSPUFJOQSPEVDUIZQFSTFOTJUJWJUZSFBDUJPOTBSFQPTTJCMFUIFSFGPSFBQQSPQSJBUFNFEJDBMTVQQPSUTIPVMECFSFBEJMZ
BWBJMBCMFXIFO713*7JTBENJOJTUFSFE*GBTFWFSFSFBDUJPOPDDVSTDVSSFOUNFEJDBMTUBOEBSETGPSFNFSHFODZUSFBUNFOUBSFUPCF
followed.
5SFBUNFOUXJUI713*7TIPVMECFBQQSPBDIFEXJUIDBVUJPOJOQBUJFOUTXIPIBWFFYIJCJUFETZNQUPNTPGIZQFSTFOTJUJWJUZUPUIFBDUJWF
JOHSFEJFOUPSFYDJQJFOUTJOUIFESVHQSPEVDUPSUPPUIFSFO[ZNFSFQMBDFNFOUUIFSBQZ
5.2 Infusion-related Reactions
Infusion-related reactions were the most commonly observed adverse reactions in patients treated with VPRIV in clinical studies.
5IF NPTU DPNNPOMZ PCTFSWFE TZNQUPNT PG JOGVTJPOSFMBUFE SFBDUJPOT XFSF IFBEBDIF EJ[[JOFTT IZQPUFOTJPO IZQFSUFOTJPO
OBVTFBGBUJHVFBTUIFOJBBOEQZSFYJB(FOFSBMMZUIFJOGVTJPOSFMBUFESFBDUJPOTXFSFNJMEBOEJOUSFBUNFOUOBÓWFQBUJFOUTPOTFU
PDDVSSFENPTUMZEVSJOHUIFGJSTUNPOUITPGUSFBUNFOUBOEUFOEFEUPPDDVSMFTTGSFRVFOUMZXJUIUJNF
5IF NBOBHFNFOU PG JOGVTJPOSFMBUFE SFBDUJPOT TIPVME CF CBTFE PO UIF TFWFSJUZ PG UIF SFBDUJPO FH TMPXJOH UIF JOGVTJPO SBUF
USFBUNFOUXJUINFEJDBUJPOTTVDIBTBOUJIJTUBNJOFTBOUJQZSFUJDTBOEPSDPSUJDPTUFSPJETBOEPSTUPQQJOHBOESFTVNJOHUSFBUNFOU
with increased infusion time.
1SFUSFBUNFOU XJUI BOUJIJTUBNJOFT BOEPS DPSUJDPTUFSPJET NBZ QSFWFOU TVCTFRVFOU SFBDUJPOT JO UIPTF DBTFT XIFSF TZNQUPNBUJD
USFBUNFOUXBTSFRVJSFE1BUJFOUTXFSFOPUSPVUJOFMZQSFNFEJDBUFEQSJPSUPJOGVTJPOPG713*7EVSJOHDMJOJDBMTUVEJFT
6 ADVERSE REACTIONS
6.1 Clinical Studies Experience
5IF EBUB EFTDSJCFE CFMPX SFGMFDU FYQPTVSF PG QBUJFOUT XJUI UZQF (BVDIFS EJTFBTF XIP SFDFJWFE 713*7 BU EPTFT SBOHJOH
GSPN6OJUTLHUP6OJUTLHFWFSZPUIFSXFFLJODMJOJDBMTUVEJFT'JGUZGPVS
QBUJFOUTXFSFOBÓWFUP&35BOESFDFJWFE
713*7 GPS NPOUIT BOE QBUJFOUT TXJUDIFE GSPN JNJHMVDFSBTF UP 713*7 USFBUNFOU BOE SFDFJWFE 713*7 GPS NPOUIT <see
Clinical Studies (14)>1BUJFOUTXFSFCFUXFFOBOEZFBSTPMEBUUJNFPGGJSTUUSFBUNFOUXJUI713*7BOEJODMVEFENBMFBOE
GFNBMFQBUJFOUT
5IFNPTUTFSJPVTBEWFSTFSFBDUJPOTJOQBUJFOUTUSFBUFEXJUI713*7XFSFIZQFSTFOTJUJWJUZSFBDUJPOT<see Warnings and Precautions
(5.1)>
General disorders and administration
site conditions
"TUIFOJB'BUJHVF
%FOPUFTBOZFWFOUDPOTJEFSFESFMBUFEUPBOEPDDVSSJOHXJUIJOVQUPIPVSTPG713*7JOGVTJPO
-FTTDPNNPOBEWFSTFSFBDUJPOTBGGFDUJOHNPSFUIBOPOFQBUJFOUJOUIFUSFBUNFOUOBÓWFHSPVQBOEJOQBUJFOUTTXJUDIFE
GSPNJNJHMVDFSBTFUP713*7USFBUNFOU
XFSFCPOFQBJOUBDIZDBSEJBSBTIVSUJDBSJBGMVTIJOHIZQFSUFOTJPOBOEIZQPUFOTJPO
Pediatric Patients
"MMBEVMUBEWFSTFSFBDUJPOTUP713*7BSFDPOTJEFSFESFMFWBOUUPQFEJBUSJDQBUJFOUTBHFTUPZFBST
"EWFSTFSFBDUJPOTNPSF
DPNNPOMZTFFOJOQFEJBUSJDQBUJFOUTDPNQBSFEUPBEVMUQBUJFOUTJODMVEFEJGGFSFODF
VQQFSSFTQJSBUPSZUSBDUJOGFDUJPOSBTI
B155QSPMPOHFEBOEQZSFYJB
Immunogenicity
"TXJUIBMMUIFSBQFVUJDQSPUFJOTUIFSFJTBQPUFOUJBMGPSJNNVOPHFOJDJUZ*ODMJOJDBMTUVEJFTPGUSFBUNFOUOBÓWFQBUJFOUTUSFBUFE
XJUI713*7EFWFMPQFE*H(DMBTTBOUJCPEJFTUP713*7*OUIJTQBUJFOUUIFBOUJCPEJFTXFSFEFUFSNJOFEUPCFOFVUSBMJ[JOHJOBOJO
WJUSPBTTBZ/PJOGVTJPOSFMBUFESFBDUJPOTXFSFSFQPSUFEGPSUIJTQBUJFOU*UJTVOLOPXOJGUIFQSFTFODFPG*H(BOUJCPEJFTUP713*7
JTBTTPDJBUFEXJUIBIJHIFSSJTLPGJOGVTJPOSFBDUJPOT1BUJFOUTXJUIBOJNNVOFSFTQPOTFUPPUIFSFO[ZNFSFQMBDFNFOUUIFSBQJFT
who are switching to VPRIV should continue to be monitored for antibodies.
*NNVOPHFOJDJUZ BTTBZ SFTVMUT BSF IJHIMZ EFQFOEFOU PO UIF TFOTJUJWJUZ BOE TQFDJGJDJUZ PG UIF BTTBZ "EEJUJPOBMMZ UIF PCTFSWFE
JODJEFODFPGBOUJCPEZQPTJUJWJUZJOBOBTTBZNBZCFJOGMVFODFECZTFWFSBMGBDUPSTJODMVEJOHBTTBZNFUIPEPMPHZTBNQMFIBOEMJOH
UJNJOHPGTBNQMFDPMMFDUJPODPODPNJUBOUNFEJDBUJPOTBOEVOEFSMZJOHEJTFBTF'PSUIFTFSFBTPOTDPNQBSJTPOPGUIFJODJEFODFPG
antibodies to VPRIV with the incidence of antibodies to other products may be misleading.
7 DRUG INTERACTIONS
/PESVHESVHJOUFSBDUJPOTUVEJFTIBWFCFFODPOEVDUFE
8 USE IN SPECIFIC POPULATIONS
8.1 Pregnancy – Category B
3FQSPEVDUJPO TUVEJFT XJUI WFMBHMVDFSBTF BMGB IBWF CFFO QFSGPSNFE JO QSFHOBOU SBUT BU JOUSBWFOPVT EPTFT VQ UP NHLHEBZ
NHNEBZBCPVUUJNFTUIFSFDPNNFOEFEIVNBOEPTFPG6OJUTLHEBZPSNHLHEBZPSNHNEBZCBTFE
POUIFCPEZTVSGBDFBSFB
3FQSPEVDUJPOTUVEJFTIBWFCFFOQFSGPSNFEJOQSFHOBOUSBCCJUTBUJOUSBWFOPVTEPTFTVQUPNHLHEBZ
NHNEBZ BCPVU UJNFT UIF SFDPNNFOEFE IVNBO EPTF PG 6OJUTLHEBZ CBTFE PO UIF CPEZ TVSGBDF BSFB
5IFTF
studies did not reveal any evidence of impaired fertility or harm to the fetus due to velaglucerase alfa.
"QSFBOEQPTUOBUBMEFWFMPQNFOUTUVEZJOSBUTTIPXFEOPFWJEFODFPGBOZBEWFSTFFGGFDUPOQSFBOEQPTUOBUBMEFWFMPQNFOUBU
EPTFTVQUPNHLHNHNEBZBCPVUUJNFTUIFSFDPNNFOEFEIVNBOEPTFPG6OJUTLHEBZCBTFEPOUIFCPEZ
TVSGBDF BSFB
5IFSF BSF IPXFWFS OP BEFRVBUF BOE XFMMDPOUSPMMFE TUVEJFT JO QSFHOBOU XPNFO #FDBVTF BOJNBM SFQSPEVDUJPO
TUVEJFTBSFOPUBMXBZTQSFEJDUJWFPGIVNBOSFTQPOTF713*7TIPVMECFVTFEEVSJOHQSFHOBODZPOMZJGDMFBSMZOFFEFE
8.3 Nursing Mothers
5IFSFBSFOPEBUBGSPNTUVEJFTJOMBDUBUJOHXPNFO*UJTOPULOPXOXIFUIFSUIJTESVHJTFYDSFUFEJOIVNBONJML#FDBVTFNBOZ
ESVHTBSFFYDSFUFEJOIVNBONJMLDBVUJPOTIPVMECFFYFSDJTFEXIFO713*7JTBENJOJTUFSFEUPBOVSTJOHXPNBO
8.4 Pediatric Use
5IFTBGFUZBOEFGGFDUJWFOFTTPG713*7IBWFCFFOFTUBCMJTIFEJOQBUJFOUTCFUXFFOBOEZFBSTPGBHF6TFPG713*7JOUIJTBHF
HSPVQ JT TVQQPSUFE CZ FWJEFODF GSPN BEFRVBUF BOE XFMMDPOUSPMMFE TUVEJFT PG 713*7 JO BEVMUT BOE QFEJBUSJD < PG >
QBUJFOUT5IFTBGFUZBOEFGGJDBDZQSPGJMFTXFSFTJNJMBSCFUXFFOQFEJBUSJDBOEBEVMUQBUJFOUT<see Adverse Reactions (6.1) and Clinical
Studies (14)>5IFTBGFUZPG713*7IBTOPUCFFOFTUBCMJTIFEJOQFEJBUSJDQBUJFOUTZPVOHFSUIBOZFBSTPGBHF
8.5 Geriatric Use
%VSJOHDMJOJDBMTUVEJFTQBUJFOUTBHFEPSPMEFSXFSFUSFBUFEXJUI713*7$MJOJDBMTUVEJFTPG713*7EJEOPUJODMVEFTVGGJDJFOU
OVNCFSTPGTVCKFDUTBHFEBOEPWFSUPEFUFSNJOFXIFUIFSUIFZSFTQPOEEJGGFSFOUMZGSPNZPVOHFSTVCKFDUT0UIFSSFQPSUFEDMJOJDBM
FYQFSJFODFIBTOPUJEFOUJGJFEEJGGFSFODFTJOSFTQPOTFTCFUXFFOUIFFMEFSMZBOEZPVOHFSQBUJFOUT*OHFOFSBMEPTFTFMFDUJPOGPSBO
FMEFSMZQBUJFOUTIPVMECFBQQSPBDIFEDBVUJPVTMZDPOTJEFSJOHQPUFOUJBMDPNPSCJEDPOEJUJPOT
10 OVERDOSAGE
14.2 Study in Patients Switching from Imiglucerase Treatment to VPRIV
4UVEZ***XBTBNPOUIPQFOMBCFMTJOHMFBSNNVMUJOBUJPOBMTUVEZJOQBUJFOUTBHFZFBSTBOEPMEFSXIPIBECFFOSFDFJWJOH
USFBUNFOU XJUI JNJHMVDFSBTF BU EPTFT SBOHJOH CFUXFFO 6OJUTLH UP 6OJUTLH GPS B NJOJNVN PG DPOTFDVUJWF NPOUIT
1BUJFOUTBMTPXFSFSFRVJSFEUPIBWFBTUBCMFCJXFFLMZEPTFPGJNJHMVDFSBTFGPSBUMFBTUNPOUITQSJPSUPFOSPMMNFOU5IFNFBO
BHFXBTZFBSTBOEXFSFGFNBMF*NJHMVDFSBTFUIFSBQZXBTTUPQQFEBOEUSFBUNFOUXJUI713*7XBTBENJOJTUFSFEFWFSZ
PUIFSXFFLBUUIFTBNFOVNCFSPGVOJUTBTUIFQBUJFOUTQSFWJPVTJNJHMVDFSBTFEPTF"EKVTUNFOUPGEPTBHFXBTBMMPXFECZTUVEZ
criteria if needed in order to maintain clinical parameters.
)FNPHMPCJO DPODFOUSBUJPOT BOE QMBUFMFU DPVOUT SFNBJOFE TUBCMF PO BWFSBHF UISPVHI NPOUIT PG 713*7 USFBUNFOU "GUFS NPOUITPGUSFBUNFOUXJUI713*7UIFNFEJBOIFNPHMPCJODPODFOUSBUJPOXBTHE-SBOHF
WTUIFCBTFMJOFWBMVFPG
HE-SBOHF
BOEUIFNFEJBOQMBUFMFUDPVOUBGUFSNPOUITXBT¦-SBOHF
WTUIFCBTFMJOF
WBMVFPG¦-SBOHF
/PQBUJFOUSFRVJSFEEPTBHFBEKVTUNFOUEVSJOHUIFNPOUIUSFBUNFOUQFSJPE
15 REFERENCES
1BTUPSFT(.8FJOSFC/+"FSUT)FUBM5IFSBQFVUJD(PBMTJOUIF5SFBUNFOUPG(BVDIFS%JTFBTF4FNJO)FNBUPM
4VQQM
5IFSFJTOPFYQFSJFODFXJUIPWFSEPTFPG713*7
11 DESCRIPTION
5IFBDUJWFJOHSFEJFOUPG713*7JTWFMBHMVDFSBTFBMGBXIJDIJTQSPEVDFECZHFOFBDUJWBUJPOUFDIOPMPHZJOBIVNBOGJCSPCMBTUDFMM
MJOF7FMBHMVDFSBTFBMGBJTBHMZDPQSPUFJOPGBNJOPBDJETXJUIBNPMFDVMBSXFJHIUPGBQQSPYJNBUFMZL%B7FMBHMVDFSBTF
BMGBIBTUIFTBNFBNJOPBDJETFRVFODFBTUIFOBUVSBMMZPDDVSSJOHIVNBOFO[ZNFHMVDPDFSFCSPTJEBTF7FMBHMVDFSBTFBMGBDPOUBJOT
QPUFOUJBM/MJOLFEHMZDPTZMBUJPOTJUFTGPVSPGUIFTFTJUFTBSFPDDVQJFECZHMZDBODIBJOT7FMBHMVDFSBTFBMGBJTNBOVGBDUVSFEUP
DPOUBJOQSFEPNJOBOUMZIJHINBOOPTFUZQF/MJOLFEHMZDBODIBJOT5IFIJHINBOOPTFUZQF/MJOLFEHMZDBODIBJOTBSFTQFDJGJDBMMZ
SFDPHOJ[FE BOE JOUFSOBMJ[FE WJB UIF NBOOPTF SFDFQUPS QSFTFOU PO UIF TVSGBDF PO NBDSPQIBHFT UIF DFMMT UIBU BDDVNVMBUF
glucocerebroside in Gaucher disease. Velaglucerase alfa catalyzes the hydrolysis of the glycolipid glucocerebroside to glucose
and ceramide in the lysosome.
713*7 JT EPTFE CZ 6OJUTLH XIFSF POF 6OJU PG FO[ZNF BDUJWJUZ JT EFGJOFE BT UIF RVBOUJUZ PG FO[ZNF SFRVJSFE UP DPOWFSU POF
micromole of p-nitrophenyl ß%HMVDPQZSBOPTJEFUPQOJUSPQIFOPMQFSNJOVUFBU$
713*7JTTVQQMJFEBTBTUFSJMFQSFTFSWBUJWFGSFFMZPQIJMJ[FEQPXEFSJOTJOHMFVTFWJBMT'PMMPXJOHSFDPOTUJUVUJPOXJUI4UFSJMF8BUFS
GPS*OKFDUJPO641UIFTPMVUJPODPOUBJOTUIFDPNQPOFOUTMJTUFEJO5BCMF
Table 3: VPRIV Composition Following Reconstitution
16 HOW SUPPLIED/STORAGE AND HANDLING
713*7JTBTUFSJMFQSFTFSWBUJWFGSFFMZPQIJMJ[FEQPXEFSSFRVJSJOHSFDPOTUJUVUJPOBOEGVSUIFSEJMVUJPOQSJPSUPVTF*UJTTVQQMJFE
JOJOEJWJEVBMMZQBDLBHFEHMBTTWJBMTXIJDIBSFDMPTFEXJUIBCVUZMSVCCFSTUPQQFSXJUIBGMVPSPSFTJODPBUJOHBOEBSFTFBMFEXJUI
BOBMVNJOVNPWFSTFBMXJUIBGMJQPGGQMBTUJDDBQ5IFWJBMTBSFJOUFOEFEGPSTJOHMFVTFPOMZ713*7JTBWBJMBCMFBT6OJUTWJBM
/%$BOE6OJUTWJBM/%$
16.1 Storage
713*7TIPVMECFTUPSFEJOBSFGSJHFSBUPSBUUP$UP'
%POPUVTF713*7BGUFSUIFFYQJSBUJPOEBUFPOUIFWJBM
Do not freeze.
Protect vial from light.
17 PATIENT COUNSELING INFORMATION
r713*7TIPVMECFBENJOJTUFSFEVOEFSUIFTVQFSWJTJPOPGBIFBMUIDBSFQSPGFTTJPOBM713*7JTBUSFBUNFOUUIBUJTHJWFOJOUSBWFOPVTMZ
CZ*7
FWFSZPUIFSXFFL5IFJOGVTJPOUZQJDBMMZUBLFTVQUPNJOVUFT
r1BUJFOUT TIPVME CF BEWJTFE UIBU 713*7 NBZ DBVTF IZQFSTFOTJUJWJUZ SFBDUJPOT PS JOGVTJPOSFMBUFE SFBDUJPOT *OGVTJPOSFMBUFE
SFBDUJPOTDBOVTVBMMZCFNBOBHFECZTMPXJOHUIFJOGVTJPOSBUFUSFBUNFOUXJUINFEJDBUJPOTTVDIBTBOUJIJTUBNJOFTBOUJQZSFUJDT
BOEPSDPSUJDPTUFSPJETBOEPSTUPQQJOHBOESFTVNJOHUSFBUNFOUXJUIJODSFBTFEJOGVTJPOUJNF1SFUSFBUNFOUXJUIBOUJIJTUBNJOFT
BOEPSDPSUJDPTUFSPJETNBZQSFWFOUTVCTFRVFOUSFBDUJPOT5SFBUNFOUXJUI713*7TIPVMECFDBSFGVMMZSFFWBMVBUFEJOUIFQSFTFODF
PGTJHOJGJDBOUFWJEFODFPGIZQFSTFOTJUJWJUZUPUIFQSPEVDU<TFF8BSOJOHTBOE1SFDBVUJPOT
>
Extractable 200 Units/vial
Extractable 400 Units/vial
6OJUT
6OJUT
DJUSJDBDJENPOPIZESBUF
NH
5.04 mg
QPMZTPSCBUF
NH
0.44 mg
4IJSF)VNBO(FOFUJD5IFSBQJFT*OD
.BJO4USFFU
$BNCSJEHF."
TPEJVNDJUSBUFEJIZESBUF
NH
NH
0OF1BUIJTBTFSWJDFNBSLBOE713*7JTBUSBEFNBSLPG4IJSF)VNBO(FOFUJD5IFSBQJFT*OD
sucrose
100 mg
NH
Active Ingredient
velaglucerase alfa
Rx Only
713*7JTNBOVGBDUVSFECZ
Inactive Ingredients
12 CLINICAL PHARMACOLOGY
12.1 Mechanism of Action
(BVDIFSEJTFBTFJTBOBVUPTPNBMSFDFTTJWFEJTPSEFSDBVTFECZNVUBUJPOTJOUIF(#"HFOFXIJDISFTVMUTJOBEFGJDJFODZPGUIF
lysosomal enzyme beta-glucocerebrosidase. Glucocerebrosidase catalyzes the conversion of the sphingolipid glucocerebroside
into glucose and ceramide. The enzymatic deficiency causes an accumulation of glucocerebroside primarily in the lysosomal
DPNQBSUNFOUPGNBDSPQIBHFTHJWJOHSJTFUPGPBNDFMMTPSi(BVDIFSDFMMTu*OUIJTMZTPTPNBMTUPSBHFEJTPSEFS-4%
DMJOJDBMGFBUVSFT
BSFSFGMFDUJWFPGUIFBDDVNVMBUJPOPG(BVDIFSDFMMTJOUIFMJWFSTQMFFOCPOFNBSSPXBOEPUIFSPSHBOT5IFBDDVNVMBUJPOPG(BVDIFS
cells in the liver and spleen leads to organomegaly. Presence of Gaucher cells in the bone marrow and spleen lead to clinically
significant anemia and thrombocytopenia.
7FMBHMVDFSBTFBMGBDBUBMZ[FTUIFIZESPMZTJTPGHMVDPDFSFCSPTJEFSFEVDJOHUIFBNPVOUPGBDDVNVMBUFEHMVDPDFSFCSPTJEF
12.3 Pharmacokinetics
*OBNVMUJDFOUFSTUVEZDPOEVDUFEJOQFEJBUSJD/UPZFBSTPME
BOEBEVMU/UPZFBSTPME
QBUJFOUTXJUIUZQF
(BVDIFSEJTFBTFQIBSNBDPLJOFUJDFWBMVBUJPOTXFSFQFSGPSNFEBU8FFLTBOEGPMMPXJOHNJOVUFJOUSBWFOPVTJOGVTJPOTPG
713*76OJUTLHFWFSZPUIFSXFFL4FSVNWFMBHMVDFSBTFBMGBDPODFOUSBUJPOTEFDMJOFESBQJEMZXJUIBNFBOIBMGMJGFPGUP
NJOVUFT5IFNFBOWFMBHMVDFSBTFBMGBDMFBSBODFSBOHFEGSPNUPN-NJOLH5IFNFBOWPMVNFPGEJTUSJCVUJPOBUTUFBEZ
TUBUFSBOHFEGSPNUPN-LHUPPGCPEZXFJHIU
)PXFWFSCFDBVTFBOJOBEFRVBUFMZWBMJEBUFEBOBMZUJDBMBTTBZ
NFUIPEXBTVTFEJOUIFFWBMVBUJPOTUIFBDDVSBUFBOEEFGJOJUJWFQIBSNBDPLJOFUJDQBSBNFUFSWBMVFTBSFOPUDVSSFOUMZBWBJMBCMF
/PBDDVNVMBUJPOPSDIBOHFJOWFMBHMVDFSBTFBMGBQIBSNBDPLJOFUJDTPWFSUJNFGSPN8FFLTUPXBTPCTFSWFEVQPONVMUJQMF
EPTJOH6OJUTLHFWFSZPUIFSXFFL
#BTFEPOUIFMJNJUFEEBUBUIFSFXFSFOPOPUBCMFQIBSNBDPLJOFUJDEJGGFSFODFTCFUXFFONBMFBOEGFNBMFQBUJFOUTJOUIJTTUVEZ5IF
FGGFDUPGBHFPOQIBSNBDPLJOFUJDTPGWFMBHMVDFSBTFBMGBXBTJODPODMVTJWF
5IFFGGFDUPGBOUJESVHBOUJCPEZGPSNBUJPOPOUIFQIBSNBDPLJOFUJDQBSBNFUFSTPGWFMBHMVDFSBTFBMGBJTVOLOPXO
13 NONCLINICAL TOXICOLOGY
13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility
-POHUFSN TUVEJFT JO BOJNBMT UP FWBMVBUF DBSDJOPHFOJD QPUFOUJBM PS TUVEJFT UP FWBMVBUF NVUBHFOJD QPUFOUJBM IBWF OPU CFFO
performed with velaglucerase alfa.
*O B NBMF BOE GFNBMF GFSUJMJUZ TUVEZ JO SBUT WFMBHMVDFSBTF BMGB EJE OPU DBVTF BOZ TJHOJGJDBOU BEWFSTF FGGFDU PO NBMF PS GFNBMF
GFSUJMJUZQBSBNFUFSTVQUPBNBYJNVNEPTFPGNHLHEBZNHNEBZBCPVUUJNFTUIFSFDPNNFOEFEIVNBOEPTFPG
6OJUTLHEBZCBTFEPOUIFCPEZTVSGBDFBSFB
14 CLINICAL STUDIES
5IFFGGJDBDZPG713*7XBTBTTFTTFEJOUISFFDMJOJDBMTUVEJFTJOBUPUBMPGQBUJFOUTXJUIUZQF(BVDIFSEJTFBTFQBUJFOUTBHF
ZFBSTBOEPMEFSSFDFJWFE713*7BOEQBUJFOUTBHFZFBSTBOEPMEFSSFDFJWFEJNJHMVDFSBTF4UVEJFT*BOE**XFSFDPOEVDUFEJO
patients who were not currently receiving Gaucher disease-specific therapy. Study III was conducted in patients who were receiving
JNJHMVDFSBTFUSFBUNFOUJNNFEJBUFMZCFGPSFTUBSUJOH713*7*OUIFTFTUVEJFT713*7XBTBENJOJTUFSFEJOUSBWFOPVTMZPWFSNJOVUFT
BUEPTFTSBOHJOHGSPN6OJUTLHUP6OJUTLHFWFSZPUIFSXFFL
14.1 Studies of VPRIV as Initial Therapy
4UVEZ*XBTBNPOUISBOEPNJ[FEEPVCMFCMJOEQBSBMMFMEPTFHSPVQNVMUJOBUJPOBMTUVEZJOQBUJFOUTBHFZFBSTBOEPMEFS
with Gaucher disease-related anemia and either thrombocytopenia or organomegaly. Patients were not allowed to have had diseaseTQFDJGJDUIFSBQZGPSBUMFBTUUIFQSFWJPVTNPOUITBMMCVUPOFIBEOPQSJPSUIFSBQZ5IFNFBOBHFXBTZFBSTBOEXFSFNBMF
1BUJFOUTXFSFSBOEPNJ[FEUPSFDFJWF713*7BUBEPTFPGFJUIFS6OJUTLH/
PS6OJUTLH/
FWFSZPUIFSXFFL
"U CBTFMJOF NFBO IFNPHMPCJO DPODFOUSBUJPO XBT HE- NFBO QMBUFMFU DPVOU XBT Y - NFBO MJWFS WPMVNF XBT PGCPEZXFJHIU#8
BOENFBOTQMFFOWPMVNFXBT#8'PSBMMTUVEJFTMJWFSBOETQMFFOWPMVNFTXFSFNFBTVSFECZ.3*
5IFDIBOHFTJODMJOJDBMQBSBNFUFSTBGUFSNPOUITPGUSFBUNFOUBSFTIPXOJO5BCMF5IFPCTFSWFEDIBOHFGSPNCBTFMJOFJOUIF
QSJNBSZFOEQPJOUIFNPHMPCJODPODFOUSBUJPOXBTDPOTJEFSFEUPCFDMJOJDBMMZNFBOJOHGVMJOMJHIUPGUIFOBUVSBMIJTUPSZPGVOUSFBUFE
Gaucher disease.
Table 4: Mean Change from Baseline to Month 12 for Clinical Parameters in Patients with Type 1 Gaucher Disease Initiating
Therapy with VPRIV in Study I
Mean Changes from Baseline ± Std. Err. of the Mean
VPRIV Dose (given every other week)
Clinical Parameter
45 Units/kg
N = 13
60 Units/kg
N = 12
)FNPHMPCJODPODFOUSBUJPODIBOHF
HE-
š
š
Platelet count change
Y-
š
š
-JWFSWPMVNFDIBOHF
#8
š
š
Spleen volume change
#8
š
š
1SJNBSZTUVEZFOEQPJOUXBTIFNPHMPCJODPODFOUSBUJPODIBOHFJOUIF6OJULHHSPVQQ
* Statistically significant changes from baseline after adjusting for performing multiple tests
4UVEZ ** XBT B NPOUI SBOEPNJ[FE EPVCMFCMJOE BDUJWFDPOUSPMMFE JNJHMVDFSBTF
QBSBMMFMHSPVQ NVMUJOBUJPOBM TUVEZ JO QBUJFOUTBHFZFBSTBOEPMEFS1BUJFOUTXFSFSFRVJSFEUPIBWF(BVDIFSEJTFBTFSFMBUFEBOFNJBBOEFJUIFSUISPNCPDZUPQFOJBPS
PSHBOPNFHBMZ1BUJFOUTXFSFOPUBMMPXFEUPIBWFIBEEJTFBTFTQFDJGJDUIFSBQZGPSBUMFBTUUIFQSFWJPVTNPOUIT5IFNFBOBHF
XBTZFBSTBOEXFSFGFNBMFUIFZPVOHFTUQBUJFOUXIPSFDFJWFE713*7XBTBHFZFBST1BUJFOUTXFSFSBOEPNJ[FEUP
SFDFJWFFJUIFS6OJUTLHPG713*7/
PS6OJUTLHPGJNJHMVDFSBTF/
FWFSZPUIFSXFFL
"UCBTFMJOFUIFNFBOIFNPHMPCJODPODFOUSBUJPOXBTHE-NFBOQMBUFMFUDPVOUXBTY-BOENFBOMJWFSWPMVNFXBT
#8'PSUIFQBUJFOUTXIPIBEOPUIBETQMFOFDUPNZJOFBDIHSPVQ
UIFNFBOTQMFFOWPMVNFXBT#8"GUFSNPOUIT
PGUSFBUNFOUUIFNFBOBCTPMVUFJODSFBTFGSPNCBTFMJOFJOIFNPHMPCJODPODFOUSBUJPOXBTHE-š4&
GPSQBUJFOUTUSFBUFE
XJUI713*75IFNFBOUSFBUNFOUEJGGFSFODFJODIBOHFGSPNCBTFMJOFUPNPOUIT<713*7JNJHMVDFSBTF>XBTHE-š4&
*O 4UVEJFT * BOE ** FYBNJOBUJPO PG BHF BOE HFOEFS TVCHSPVQT EJE OPU JEFOUJGZ EJGGFSFODFT JO SFTQPOTF UP 713*7 BNPOH UIFTF
TVCHSPVQT5IFOVNCFSPGOPO$BVDBTJBOQBUJFOUTJOUIFTFTUVEJFTXBTUPPTNBMMUPBEFRVBUFMZBTTFTTBOZEJGGFSFODFJOFGGFDUT
by race.