WARNING: RISK OF ANAPHYLAXIS
PALYNZIQ can cause a severe allergic reaction (anaphylaxis) that may be life-threatening and can happen any time during treatment with PALYNZIQ.
For more information, read the Important Safety Information below or see the full Prescribing Information at PALYNZIQ.com.
Current Studies
Study 165-306 (PEGASUS)
RecruitingA Phase 3 Multi Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12 17) with Phenylketonuria Featuring an Open Label Randomized Two Arm (Active vs Diet Only Control) Design
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Indication
Phenylketonuria
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Investigational therapeutic
BMN165
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Study Type
Phase 3
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Goal
Evaluate safety and efficacy of BMN 165 in adolescents with PKU when administered as an Induction/Titration and Maintenance dose regimen.
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Status
Recruiting
Study 165-305
A Phase 3, Open-Label, Multi-Center Study to Assess the Safety and Efficacy of BMN 165 in Japanese Subjects 18 Years of Age and Older With Phenylketonuria
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Indication
Phenylketonuria
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Investigational therapeutic
BMN165
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Study Type
Phase 3
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Goal
The primary objective of the study is to evaluate the efficacy and safety of BMN 165 in Japanese subjects ≥ 18 years of age with PKU when administered as an Induction/Titration and Maintenance dose regimen.
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Status
This study is ongoing, but not recruiting participants.
Study 165-508
RecruitingA Multicentre, Observational Study to Evaluate the Real-World Outcomes of Palynziq® (Pegvaliase) in Subjects with Phenylketonuria in Germany
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Indication
Phenylketonuria (PKU)
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Investigational therapeutic
Palynziq® (pegvaliase)
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Study Type
Phase 4
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Goal
In addition to the efficacy and safety profile of pegvaliase that has been established in clinical trials; there remains an important need to provide data to prescribers and the phenylketonuria (PKU) community on the real-world usage of pegvaliase, and associated outcomes, to support clinical practice optimization and provide greater understanding of important clinical issues in PKU
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Status
This study is currently recruiting participants
Study 165-501
RecruitingA Multi-Center, Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq® (pegvaliase) in Subjects with Phenylketonuria
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Indication
Phenylketonuria (PKU)
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Investigational Therapeutic
N/A
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Study Type
Phase 4
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Goal
– To quantify and characterize the risk of protocol-defined safety events in incident-users receiving pegvaliase for the treatment of PKU in a real-world setting.
– To quantify and characterize the risk of protocol-defined safety events in subjects receiving pegvaliase for the treatment of PKU in a real-world setting.
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Status
This study is currently recruiting participants
Study 165-504
RecruitingA Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
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Indication
Phenylketonuria (PKU)
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Investigational Therapeutic
Pegvaliase
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Study Type
Phase 4
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Goal
This is a Phase 4 observational study designed to assess the impact of Palynziq ® (pegvaliase) treatment in pregnant women with PKU and on their offspring who were exposed to pegvaliase at any time during pregnancy and breastfeeding
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Status
This study is currently recruiting participants
Completed Studies
Study 165-304
An Open-label Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase (> 40mg/day Dose) in Adults with Phenylketonuria.
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Indication
Phenylketonuria (PKU)
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Investigational Therapeutic
PALYNZIQ® (pegvaliase, rAvPAL-PEG or BMN 165)
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Study Type
Phase 3
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Goal
Evaluate long-term safety and efficacy of Pegvaliase in (> 40mg/day dose) in adults with PKU
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Status
This study has been completed
Study 165-302
A Four-Part, Phase 3, Randomized, Double-Blind, Placebo-Controlled, Four-Arm, Discontinuation Study to Evaluate the Efficacy and Safety of Subcutaneous Injections of BMN 165 Self Administered by Adults with Phenylketonuria
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Indication
Phenylketonuria (PKU)
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Investigational Therapeutic
PALYNZIQ® (pegvaliase, rAvPAL-PEG or BMN 165)
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Study Type
Phase 3
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Goal
The BMN 165 clinical development program has been designed to demonstrate the safety and efficacy of BMN 165 in reducing blood Phe concentrations in adults with PKU
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Status
This study has been completed
Study PAL-003
Long-term Extension of a Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous Doses of rAvPAL-PEG in Subjects with PKU
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Indication
Phenylketonuria (PKU)
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Investigational Therapeutic
PALYNZIQ® (pegvaliase, rAvPAL-PEG or BMN 165)
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Study Type
Phase 2
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Goal
Evaluate the long-term safety, tolerability and effectiveness of injections of an investigational drug for people with PKU who have trouble controlling and maintaining their Phe levels
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Status
This study has been completed
Study 165-205
A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients with Phenylketonuria for 24 Weeks
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Indication
Phenylketonuria (PKU)
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Investigational Therapeutic
PALYNZIQ® (pegvaliase, rAvPAL-PEG or BMN 165)
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Study Type
Phase 2
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Goal
Evaluate the effect of dosing regimens of multiple subcutaneous (SC) doses of rAvPAL-PEG to induce an early and sustained Phe reduction while decreasing the frequency and severity of hypersensitivity reactions in patients with PKU
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Status
This study has been completed
Study PAL-004
A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Dose Levels of Ravpal-PEG Administered Daily in Subjects with Phenylketonuria
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Indication
Phenylketonuria (PKU)
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Investigational Therapeutic
PALYNZIQ® (pegvaliase, rAvPAL-PEG or BMN 165)
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Study Type
Phase 2
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Goal
Evaluate the effect of daily administration of rAvPAL-PEG on the reduction of blood Phe concentrations in subjects with PKU
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Status
This study has been completed
Study 165-301
A Phase 3, Open-Label, Randomized, Multi-Center Study to Assess the Safety and Tolerability of an Induction, Titration, and Maintenance Dose Regimen of Pegvaliase Self-Administered by Adults with Phenylketonuria Not Previously Treated with Pegvaliase
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Indication
Phenylketonuria (PKU)
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Investigational Therapeutic
PALYNZIQ® (pegvaliase, rAvPAL-PEG or BMN 165)
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Study Type
Phase 3
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Goal
Evaluate the safety and tolerability of injections of an investigational drug (also called PEG PAL) for people with PKU who have not received Pegvaliase in the past
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Status
This study has been completed