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Kuvan

Phase 4FDA approved 2007

Phenylketonuria | Small molecule | Metabolic |Merck KGaA|Trials Updated: Jul 28, 2014

Kuvan development status

FDA approval Dec 13, 2007
Approval holderBiomarin Pharm
ApplicationNDA022181
Highest phase Phase 4 (NCT01082328)
Phase scored for MKGAFPhase 3
Registered trials 25 across 9 sponsors since Feb 2005

Label indication KUVAN ® is indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). KUVAN is to be used in conjunction with a Phe-restricted diet. FDA label

Kuvan target and mechanism

ModalitySmall molecule

Also known as Sapropterin dihydrochloride, 6R-BH4 tetrahydrobiopterin

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment90

FDA Designations

No designations recorded

Kuvan clinical trials

Kuvan · 1 trial · 1 indication

Phase 3 1
NCT01732471Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With PhenylketonuriaPhenylketonuria
COMPLETED90 Analytics
PHASE3COMPLETED
Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria
Phenylketonuria
NCT0173247190
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Study Endpoints

Primary Endpoints

Percentage of Participants With Response to Kuvan® (Sapropterin Dihydrochloride) Treatment
Day 8

Response to Kuvan® (sapropterin dihydrochloride) treatment was defined as a reduction in blood phenylalanine levels of greater than or equal to 30% at Day 8 as compared to baseline.

Secondary Endpoints

Percent Change From Baseline in Blood Phenylalanine Levels at Day 8 in Overall Population
Baseline, Day 8
Percent Change From Baseline in Blood Phenylalanine Levels at Day 8 in Sub-population of Responders
Baseline, Day 8
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) in Overall Safety Population
Baseline up to Week 11
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Kuvan®EXPERIMENTAL -

Interventions

NameTypeDescription
Kuvan®DRUGKuvan® (sapropterin dihydrochloride) will be administered orally at a dose of 20 milligram per kilogram per day (mg/kg/day) once daily for 8 days. If there is 30 percent (%) decrease in blood phenylalanine levels from baseline at the end of Day 8, then treatment will be continued at the same dose for further 6 weeks.
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Eligibility Criteria

Age Range4 Years to 18 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Willing and able to provide written informed consent (for children under 18 years old the parent\[s\]/guardians give informed consent, subjects 14-17 years old give additionally their own written informed consent) * Age of 4 - 18 years, inclusive * Confirmed clinical and bioch...

Countries:Germany
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Competitive Landscape -Phenylketonuria 14 trials

Top 5 of 7 competitors

CompanyTickerTrialsLead PhaseDrugs
BioMarin Pharmaceutical Inc.BMRN5PHASE3Pegvaliase, BMN 307
PTC Therapeutics, Inc.PTCT1PHASE3Sepiapterin
Maze Therapeutics, Inc.MAZE1PHASE2MZE782
Sanofi SA Sponsored ADRSNY1PHASE1SAR444836
Agios Pharmaceuticals, Inc.AGIO1PHASE1AG-181
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