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Idursulfase

Phase 2

Hunter Syndrome | Monoclonal antibody | Rare Disease |Takeda Pharmaceutical Company Limited|Last Updated: Jun 10, 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
UNCONTROLLEDDMC
Total Trials1
Total Enrollment94
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00630747Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving IdursulfasePHASE2 COMPLETED 94 — — Sep 13, 2004Jan 31, 2008Jun 10, 202152 United States, Brazil +8
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Study Endpoints
Primary Endpoints
Change From Baseline in Mean Percent Predicted Forced Vital Capacity (FVC) at Week 105
Baseline and at Week 105

Determined by spirometry. The change is calculated as Week 105 minus baseline.

Change From Baseline in Mean Distance Walked in the 6-minute Walk Test (6MWT) at Week 105
Baseline and at Week 105

Determined on a walking course. The change was calculated as Week 105 minus baseline.

Secondary Endpoints
Change From Baseline in Mean Passive Joint Range of Motion (JROM) at Week 105
Baseline and at Week 105
Change From Baseline in Mean Combined Liver and Spleen Volume at Week 105
Baseline and at Week 105
Change From Baseline in Mean Normalized Urine Glycosaminoglycans (GAG) Levels at Week 105
Baseline and at Week 105
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
IdursulfaseEXPERIMENTAL -
Interventions
NameTypeDescription
IdursulfaseBIOLOGICALSolution for intravenous infusion, 0.5 mg/kg once-weekly
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Eligibility Criteria
Age Range5 Years — N/A
SexMALE
Healthy VolunteersNo
Study Sites52

Inclusion Criteria: * Patient must have completed the double-blind phase of Study TKT024, defined as completing the Week 53 final evaluations. * Patient, patient's parent(s), or legally authorized representative must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Com...

Countries:United StatesBrazilCanadaFranceGermanyItalyRomaniaSpainSwedenUnited Kingdom
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