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Pegvaliase (BMN 165) and Kuvan® for PKU

Current Clinical Trials: PKU

Study: 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: PKU

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 is ongoing, but not recruiting participants

Additional information: View on Clinicaltrials.gov

Study: 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

Investigational Therapeutic: Pegvaliase (rAvPAL-PEG or BMN 165)

Study Type: Phase 2

Goal: This study is evaluating 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 is ongoing, but not recruiting participants

Additional information: View on Clinicaltrials.gov

Study: PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

Indication: Phenylketonuria, Hyperphenylalaninaemia

Investigational Therapeutic: Kuvan

Study Type: Observational

Goal: The objective of this study is to evaluate the safety of long-term treatment with Kuvan

Status: This study is currently recruiting participants

Additional information: View on Clinicaltrials.gov

Completed Clinical Studies: PKU

Study: Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

Indication: Phenylketonuria

Investigational Therapeutic: Kuvan (formerly Phenoptin™)

Study Type: Phase 3

Goal: The primary objective of this study is to evaluate the efficacy of Phenoptin™ (sapropterin dihydrochloride) in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria

Status: This study has been completed

Additional information: View on Clinicaltrials.gov

Study: A Double-blind, Placebo-controlled, Randomized Study to Evaluate the Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Subjects with Phenylketonuria

Indication: Phenylketonuria

Investigational Therapeutic: Kuvan (formerly Phenoptin™)
 
Study Type: Phase 3

Goal: This double-blind, placebo-controlled, randomized study is designed to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU

Status: This study has been completed

Additional information: View on Clinicaltrials.gov

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

Indication: Phenylketonuria

Investigational Therapeutic: Pegvaliase (rAvPAL-PEG or BMN 165)

Study Type: Phase 2

Goal: The primary objective of the study is to 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

Additional information: View on Clinicaltrials.gov

Study: 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

Investigational Therapeutic: Pegvaliase (rAvPAL-PEG or BMN 165)

Study Type: Phase 2

Goal: The purpose of this study is to 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

Additional information: View on Clinicaltrials.gov

Study: 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

Investigational Therapeutic: Pegvaliase (rAvPAL-PEG or BMN 165)

Study Type: Phase 3

Goal: This study is to 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

Additional information: View on Clinicaltrials.gov

Clinical Trial

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.
Read more »

Clinical Trial

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
Read more »

Clinical Trial

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
Read more »

Clinical Trial

PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
Read more »

Clinical Trial

Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
Read more »

Clinical Trial

A Double-blind, Placebo-controlled, Randomized Study to Evaluate the Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Subjects with Phenylketonuria
Read more »

Clinical Trial

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

Clinical Trial

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
Read more »

BioMarin

Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI) PKU, LEMS

Products

Vimizim®
Kuvan®
Naglazyme®
Aldurazyme®
Firdapse®

Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361
E-mail: support@biomarin-rareconnections.com

Global Medical Information

Contact Global Medical Information

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