Product Description
Pegvaliase is a pegylated PAL enzyme that converts phenylalanine to ammonia and trans-cinnamic acid. (Sourced from: https://pubmed.ncbi.nlm.nih.gov/31258325/)
Mechanisms of Action: Phenylalanine Converter
Novel Mechanism: No
Modality: Peptide/Protein
Route of Administration: Subcutaneous
FDA Designation: *
Approval Status: Approved
Approved Countries: Australia | Austria | Belgium | Croatia | Czech | Estonia | European Medicines Agency | Finland | Germany | Hungary | Iceland | Ireland | Italy | Latvia | Lithuania | Luxembourg | Netherlands | Poland | Portugal | Slovakia | Slovenia | Spain | Sweden | Taiwan | United States
Approved Indications: None
Known Adverse Events: None
Company: Biomarin
Company Location: NOVATO CA 94949
Company CEO: Jean-Jacques Bienaimé
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Germany, Japan, United States
Active Clinical Trial Count: 4
Recent & Upcoming Milestones
- FDA accepted BioMarin's palynziq supplementary BLA for adolescents with PKU, with PDUFA target action date set for Feb. 28, 2026.
- Clinical Outcomes Reported - Biomarin presented P3 Phenylketonuria, Maternal results on 2025-04-02 for Pegvaliase
- Clinical Outcomes Reported - Biomarin announced they will present P3 Phenylketonuria, Maternal|Phenylketonurias results in 3Q25 for Pegvaliase
Highest Development Phases
Phase 3: Phenylketonuria, Maternal|Phenylketonurias
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
2024-510875-39-00 |
165-306 | P3 |
Active, not recruiting |
Phenylketonuria, Maternal |
2027-08-14 |
2025-05-02 |
Treatments |
|
NCT05270837 |
PEGASUS | P3 |
Active, not recruiting |
Phenylketonurias |
2025-01-14 |
31% |
2025-09-13 |
Primary Endpoints |
JapicCTI-194642 |
JapicCTI-194642 | P3 |
Active |
Phenylketonuria, Maternal |
2024-01-31 |
|||
jRCT2080224573 |
jRCT2080224573 | P3 |
Completed |
Phenylketonuria, Maternal |
2024-01-31 |