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Kuvan

Phase 3

Phenylketonuria | Small molecule | Rare Disease |BioMarin Pharmaceutical Inc.|Last Updated: Sep 15, 2017

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
RandomizedCONTROLLEDBiomarker
Total Trials1
Total Enrollment56
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01376908Kuvan® in Phenylketonuria Patients Less Than 4 Years OldPHASE3 COMPLETED 56 — — Jun 1, 2011Feb 17, 2017Sep 15, 201722 Austria, Belgium +7
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Study Endpoints
Primary Endpoints
Dietary Phenylalanine (Phe) Tolerance at Week 26
Week 26

Phe tolerance was defined as the amount of dietary Phe prescribed (milligram per kilogram per day \[mg/kg/day\]) while maintaining blood Phe levels within the selected therapeutic target range (defined as greater than or equal to \[\>=\] 120 to less than \[\<\] 360 micromoles per liter \[mcmol/L\]).

Secondary Endpoints
Mean Blood Phe Levels
Baseline, Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22, 24, and 26
Change From Baseline in Dietary Phe Tolerance After 26 Weeks
Baseline and at Week 26 (last observation carried-forward [LOCF])
Number of Subjects With Any TEAEs, AEs Related to Kuvan, Serious AEs, AEs Leading to Death, and AEs Leading to Discontinuation
From the first dose of study drug administration up to 31 days after the last dose of study drug administration
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Kuvan® + Phe-restricted dietEXPERIMENTALSubjects will be treated with Kuvan® tablets once daily along with Phe-restricted diet therapy.
Phe-restricted diet aloneOTHERSubjects will follow a Phe-restricted diet alone.
Interventions
NameTypeDescription
Kuvan®DRUGKuvan® (sapropterin dihydrochloride) tablets will be administered orally at the dose of 10 mg/kg/day and will be escalated to 20 mg/kg/day if after 4 weeks a subject's Phe tolerance is not increased by at least 20% versus baseline.
Phenylalanine (Phe)-restricted dietOTHERPhe intake will be adjusted every 2 weeks, based on the mean Phe levels of the previous 2 weeks using pre-defined Phe adjustment criteria.
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Eligibility Criteria
Age RangeN/A — 4 Years
SexALL
Healthy VolunteersNo
Study Sites22

Inclusion Criteria: * Male or female PKU infants and young children less than (\<) 4 years of age at the scheduled Day 1 visit of the 26-week study period (taking into consideration the maximum of 21 days in the screening period) * Confirmed clinical and biochemical PKU, including at least two prev...

Countries:AustriaBelgiumCzechiaGermanyItalyNetherlandsSlovakiaTurkey (Türkiye)United Kingdom
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Competitive Landscape -Phenylketonuria 12 trials
CompanyTickerTrialsLead PhaseDrugs
BioMarin Pharmaceutical Inc.BMRN5PHASE3Pegvaliase, BMN 307
PTC Therapeutics, Inc.PTCT2PHASE3PTC923, Sepiapterin
Sanofi SA Sponsored ADRSNY1PHASE1SAR444836
Agios Pharmaceuticals, Inc.AGIO1PHASE1AG-181
Illumina, Inc.ILMN1 — Undisclosed
Q32 Bio IncQTTB1 — HMI-102
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