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velaglucerase alfa

Phase 3

Gaucher Disease, Type 1 | Monoclonal antibody | Metabolic |Takeda Pharmaceutical Company Limited|Last Updated: Jun 8, 2021

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindACTIVE_CONTROLLEDBiomarker
Total Trials1
Total Enrollment34
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00553631Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher DiseasePHASE3 COMPLETED 34 — — Jan 29, 2008May 5, 2009Jun 8, 202111 United States, Argentina +7
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Study Endpoints
Primary Endpoints
Mean Change From Baseline to Month 9 in Hemoglobin (Hgb) Concentration for Each Treatment Group.
Baseline to Month 9
Secondary Endpoints
Change From Baseline to Month 9 in Platelet Counts for Each Treatment Group.
Baseline to Month 9
Change From Baseline to Month 9 in Normalized Liver Volume (Percent (%) Body Weight) for Each Treatment Group.
Baseline to Month 9
Change From Baseline to Month 9 in Normalized Spleen Volume (Percent (%) Body Weight) for Each Treatment Group.
Baseline to Month 9
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
GA-GCBEXPERIMENTALVPRIV™ ,velaglucerase alfa
imigluceraseACTIVE_COMPARATOR -
Interventions
NameTypeDescription
velaglucerase alfaBIOLOGICALIV infusion, 60 U/kg every other week for 9 months
imigluceraseBIOLOGICALIV infusion, 60 U/kg every other week for 9 months
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Eligibility Criteria
Age Range2 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites11

Inclusion Criteria Includes: * The patient has a documented diagnosis and clinical manifestation of type 1 Gaucher disease * The patient is at least 2 years of age. * The patient has not received treatment for Gaucher disease (investigational products, miglustat, or imiglucerase) within 12 months ...

Countries:United StatesArgentinaIndiaIsraelParaguayRussiaSpainTunisiaUnited Kingdom
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