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Myozyme

Phase 3

Pompe Disease (Late-onset) | Monoclonal antibody | Metabolic |Sanofi|Last Updated: Feb 6, 2014

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
UNCONTROLLEDBiomarker
Total Trials2
Total Enrollment10
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00268944Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory SupportPHASE3 COMPLETED 5 — — Dec 1, 2005Jun 1, 2007Feb 5, 20141 France
NCT00250939A Study of rhGAA in Patients With Late-Onset Pompe DiseasePHASE2 COMPLETED 5 — — Feb 1, 2005Nov 1, 2006Feb 6, 20141 Netherlands
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Study Endpoints
Primary Endpoints
Treatment effect on muscle strength and functional status.
six months and one year
Treatment effect on pulmonary function and/or ventilation conditions.
six months and one year
Treatment effect on cardiomyopathy noted at inclusion
six months and one year
Treatment effect on fatigue.
six months and one year
Treatment effect on quality of life.
six months and one year
Treatment effect on muscular atrophy.
six months and one year
Overall patient satisfaction with treatment (visual analog scale).
six months and one year
Pharmacodynamics assessment.
six months and one year
safety and PK profile rhGAA
74 weeks
FVC
74 weeks
MMT
74 weeks
Effect of treatment on muscle function
74 weeks
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
1EXPERIMENTAL -
Interventions
NameTypeDescription
MyozymeBIOLOGICAL20 mg/kg qow
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * male or female aged greater than or equal to 18 years * patient's legally authorized guardian(s) must provide signed, informed consent prior to initiation of study; patient's signature required if patient understands informed consent * patient must have a documented deficit in...

Countries:FranceNetherlands
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