A new DRUG TRIALS SNAPSHOT is now available
PALYNZIQ is a drug used to lower blood levels of phenylalanine in adults with phenylketonuria (PKU), who have high phenylalanine blood levels on current treatment. Phenylketonuria is a rare inherited disorder that, if left untreated, causes brain damage and disability because of buildup of phenylalanine in the body.
PALYNZIQ is injected under the skin (subcutaneous). The initial dose is injected once weekly for four weeks. Afterwards, the dose is adjusted following a special schedule.
Drug Trial Snapshot: PALYNZIQ
HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the clinical trials that supported the FDA approval of this drug, and whether there were differences among sex, race and age groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.
The information provided in Snapshots highlights who participated in the clinical trials that supported the FDA approval of this drug, and whether there were differences among sex, race and age groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.
LIMITATIONS OF THIS SNAPSHOT:
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your health provider about the risks and benefits of a drug. Refer to the PALYNZIQ Package Insert for complete information.
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your health provider about the risks and benefits of a drug. Refer to the PALYNZIQ Package Insert for complete information.
PALYNZIQ (pegvaliase-pqpz)
pal-lin-zeek
BioMarin Pharmaceutical, Inc.
Approval date:May 24, 2018
pal-lin-zeek
BioMarin Pharmaceutical, Inc.
Approval date:May 24, 2018
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
PALYNZIQ is a drug used to lower blood levels of phenylalanine in adults with phenylketonuria (PKU), who have high phenylalanine blood levels on current treatment.
Phenylketonuria is a rare inherited disorder that, if left untreated, causes brain damage and disability because of buildup of phenylalanine in the body.
How is this drug used?
PALYNZIQ is injected under the skin (subcutaneous). The initial dose is injected once weekly for four weeks. Afterwards, the dose is adjusted following a special schedule.
What are the benefits of this drug?
Patients treated with PALYNZIQ had lower levels of blood phenylalanine concentrations and were able to maintain low concentrations in comparison to patients who received placebo treatment.
Were there any differences in how well the drug worked in clinical trials among sex, race and age?
- Sex: PALYNZIQ worked similarly in males and females.
- Race: The majority of patients were White. The number of patients in other races was limited; therefore, differences in side effects among races could not be determined.
- Age: The majority of patients were adults 18-65 years of age; therefore, differences in response among different age groups could not be determined.
What are the possible side effects?
PALYNZIQ may cause serious side effects including life threatening allergic reaction called anaphylaxis.The most common side effects of PALYNZIQ are injection site reactions, joint pain, hypersensitivity reactions, headache, and various skin reactions.Were there any differences in side effects among sex, race and age?
- Sex: The occurrence of certain hypersensitivy side effects was higher in females than males.
- Race: The majority of patients were White. The number of patients in other races was limited; therefore, differences in side effects among races could not be determined.
- Age: The majority of patients were adults 18-65 years of age; therefore, differences in side effects among different age groups could not be determined.
WHO WAS IN THE CLINICAL TRIAL?
Who participated in the clinical trials?
The FDA approved PALYNZIQ based on evidence from 2 clinical trials, Trial 1 (NCT01819727) and Trial 2 (NCT01889862) which included 285 adult patients with phenylketonuria. The trials were conducted at 60 sites in the United States.Figure 1 summarizes how many males and females were enrolled in the clinical trials used to evaluate safety.Figure 1. Baseline Demographics by Sex (safety population)
Figure 2. Baseline Demographics by Race (safety population)
FDA ReviewTable 1. Baseline Demographics by Race (safety population)RaceNumber of PatientsPercentageWhite27898%Black or African American31%American Indian or Alaska Native1less than 1%Other2less than 1%Missing1less than 1%FDA ReviewFigure 3 summarizes the percentage of patients by age based on the safety population.Figure 3. Baseline Demographics by Age
FDA ReviewHow were the trials designed?
The benefit and side effects of PALYNZQ were evaluated in two clinical trials of adult patients with phenylketonuria (PKU). Each trial had a different design and treatment duration.Trial 1: Trial evaluated patients with high phenylalanine blood concentrations who had never been treated before with PALYNZIQ. All patients received PALYNZIQ for up to 36 weeks, starting with low doses given once weekly up to the maximum dose given once daily. The trial data were used to assess the side effects of PALYNZIQ.Trial 2: Patients, who were previously treated with PALYNZIQ, received up to an additional 13 weeks of PALYNZIQ. After that, if patients achieved at least a 20% decrease in blood phenylalanine concentration from their pre-treatment levels, they were randomly assigned to continue PALYNZIQ or to switch to placebo for 8 weeks. The benefit of PALYNZIQ was assessed by measuring the level of phenylalanine in the blood at the end of 8 weeks.GLOSSARY
CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.PRESCRIBING INFORMATION
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