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Burosumab

Phase 3

Hypophosphatemia | Monoclonal antibody | Pain |Ultragenyx Pharmaceutical Inc.|Last Updated: Jan 18, 2020

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 Trials1
Total Enrollment1
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03581591Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patient With ENS and Hypophosphatemic RicketsPHASE3 COMPLETED 1Jan 31, 2018Dec 6, 2019Jan 18, 20201 United States
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Study Endpoints
Primary Endpoints
The effect of KRN23 treatment on normalizing age-adjusted serum phosphorous levels in a single pediatric patient with Epidermal Nevus Syndrome associated hypophosphatemic rickets
18 months
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Primary; open labelEXPERIMENTALInjection of Burosumab every two to three weeks based on Serum Phosphorus level of subject. Initial dose to be 0.3 mg/kg. Subsequent dosing will be titrated up or down depending on Serum Phosphorus level for that time period.
Interventions
NameTypeDescription
BurosumabBIOLOGICALrecombinant human IgG1 monoclonal antibody to fibroblast growth factor 23)
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Eligibility Criteria
Age RangeN/A — 18 Years
SexFEMALE
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. Patient has confirmed ENS by physician diagnosis 2. Patient has confirmed FGF23 elevations in the context of low serum phosphorous \< 4.1 mg/dL 3. Patient able to tolerate KRN23 treatment 4. Have a corrected serum calcium level \< 10.8mg/dL 5. Have an eGFR \>60 ml/min 6. Must...

Countries:United States
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