Pegvaliase
Phase 2Protein / enzyme
BioMarin PharmaceuticalPegvaliase is a protein / enzyme that works as a PAL Enzyme Substitution.[2] It is being investigated across 2 indications, with its most advanced program in Phase 2.[1]
Pegvaliase is one of 28 enzyme replacement programs targeting Phenylketonuria. It is currently in Phase 2.
2
Indications
7
Total Trials
5
Active Trials
27
Competitors
Pipeline
Status by Indication[3][1]
Competition
Competitive Landscape
29 competitors in Phenylketonuria
#DrugCompanyPhaseTrials
1Avelox
BayerApproved
L-Carnitine
IIperboreal Pharma Srl
Approved
BMN 333
BioMarin PharmaceuticalPhase 2
CEB-01
BioMarin PharmaceuticalPhase 3
PTC923
PPTC Therapeutics
Phase 3
Kuvan
BioMarin PharmaceuticalPreclinical
Immunosuppression
NNational Heart, Lung, and Blood Institute (NHLBI)
Phase 2
Kuvan
BioMarin PharmaceuticalPreclinical
Tetrahydrobiopterin
OOrphanetics Pharma Entwicklungs GmbH
Phase 1
Kuvan
BioMarin PharmaceuticalPreclinical
Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.
Timeline
Expected Milestones
Clinical
Trials by Indication[1]
Pregnancy1 trial
Safety and Efficacy1 trial
Adult1 trial
Nutrition Status of Adults Treated With Pegvaliase
Completed·—··NCT03792451·18 enrolled
Sources
References
- [1]
- [2]
- [3]Open Targets Platform — Drug–disease associations for Pegvaliase. EMBL-EBI & Wellcome Sanger Institute.
- [4]
Details
Drug Info
Quick Facts
- Mechanism of Action
- PAL Enzyme Substitution[2]
- Modality
- Protein / enzyme[2]
- Highest Phase
- Phase 2[1]
- Company