© 2021 BioMarin. All rights reserved.

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 165-305

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

  • Indication

    Phenylketonuria

  • Investigational Therapeutic

    BMN165

  • Study Type

    Phase 3

  • 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.

  • Status

    This study is ongoing, but not recruiting participants.

Visit Study 165-305 Now
 

Study 165-508

Recruiting

A Multicentre, Observational Study to Evaluate the Real-World Outcomes of Palynziq® (Pegvaliase) in Subjects with Phenylketonuria in Germany

  • Indication

    Phenylketonuria (PKU)

  • Investigational therapeutic

    Palynziq® (pegvaliase)

  • Study Type

    Phase 4

  • 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

  • Status

    This study is currently recruiting participants

Visit Study 165-508 Now

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.

  • Indication

    Phenylketonuria (PKU)

  • Investigational Therapeutic

    PALYNZIQ® (pegvaliase, rAvPAL-PEG or BMN 165)

  • Study Type

    Phase 3

  • Goal

    Evaluate long-term safety and efficacy of Pegvaliase in (> 40mg/day dose) in adults with PKU

  • Status

    This study is ongoing, but not recruiting participants

Visit Study 165-304 Now
 

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

  • Indication

    Phenylketonuria (PKU)

  • Investigational Therapeutic

    PALYNZIQ® (pegvaliase, rAvPAL-PEG or BMN 165)

  • Study Type

    Phase 3

  • 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

  • Status

    This study has been completed

Visit Study 165-302 Now
 

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

  • Indication

    Phenylketonuria (PKU)

  • Investigational Therapeutic

    PALYNZIQ® (pegvaliase, rAvPAL-PEG or BMN 165)

  • Study Type

    Phase 2

  • 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

  • Status

    This study has been completed

Visit Study PAL-003 Now
 

Study 165-205

A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients with Phenylketonuria for 24 Weeks

  • Indication

    Phenylketonuria (PKU)

  • Investigational Therapeutic

    PALYNZIQ® (pegvaliase, rAvPAL-PEG or BMN 165)

  • Study Type

    Phase 2

  • 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

  • Status

    This study has been completed

Visit Study 165-205 Now
 

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

  • Indication

    Phenylketonuria (PKU)

  • Investigational Therapeutic

    PALYNZIQ® (pegvaliase, rAvPAL-PEG or BMN 165)

  • Study Type

    Phase 2

  • Goal

    Evaluate the effect of daily administration of rAvPAL-PEG on the reduction of blood Phe concentrations in subjects with PKU

  • Status

    This study has been completed

Visit Study PAL-004 Now
 

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

  • Indication

    Phenylketonuria (PKU)

  • Investigational Therapeutic

    PALYNZIQ® (pegvaliase, rAvPAL-PEG or BMN 165)

  • Study Type

    Phase 3

  • 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

  • Status

    This study has been completed

Visit Study 165-301 Now