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BMN 110

Phase 3

Mucopolysaccharidosis IVA (Morquio A Syndrome) | Small molecule | Rare Disease |BioMarin Pharmaceutical Inc.|Last Updated: Sep 26, 2019

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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment13
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01966029BMN 110 Phase 3B in Australian PatientsPHASE3 COMPLETED 13Jul 1, 2013Dec 1, 2016Sep 26, 20195 Australia
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Study Endpoints
Primary Endpoints
Safety Analysis
The study period during which all non-serious AEs and SAEs will be reported begins after informed consent is obtained and through 30 days after the last study visit or 30 days after the last drug infusion, whichever comes first.

The analyses of safety will include all patients who receive any study drug. All safety data will be summarized descriptively. All AEs will be coded using the Medical Dictionary for Regulatory Activities (MedDRA). The incidence of AEs will be summarized by System Organ Class (SOC), Preferred Term (PT), relationship to study drug, and severity. Patient listings will be provided for SAEs, deaths, and AEs leading to study drug discontinuation, or study withdrawals. All AE summaries will include only treatment-emergent AEs (TEAEs) reported during the study period (AEs that occur after the first study drug infusion). Infusion-associated AEs will be summarized by SOC, PT and severity. The following safety measures will be summarized descriptively: concomitant medications, clinical laboratory tests, vital signs, ECGs, immunogenicity results, analgesic medication use, results from routine physical examinations (including standard neurologic examinations), and cervical spine imaging.

Secondary Endpoints
Efficacy Analysis
Baseline, Week 25, Week 49 or Early Termination Visit
Efficacy Analysis (recommended)
by Week 24, Week 52 and Early Termination Visit during Extension Phase
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
TreatmentEXPERIMENTALAll patients receive treatment with BMN 110
Interventions
NameTypeDescription
BMN 110DRUGAll pateints receive treatment with BMN 110
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Eligibility Criteria
SexALL
Healthy VolunteersNo
Study Sites5

Inclusion Criteria: 1. Diagnosed with MPS IVA as confirmed by a documented GALNS enzymatic test (GALNS activity in affected range, beta-galactosidase and a second lysosomal sulfatase activity within normal range). 2. Age 12 months or older. 3. Willing and able to provide written, signed informed co...

Countries:Australia
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