A new DRUG TRIALS SNAPSHOT is now available
Drug Trial Snapshot: ONPATTRO
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 ONPATTRO 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 ONPATTRO Package Insert for complete information.
ONPATTRO (patisiran)
on-PAH-troh
Alnylam Pharmaceuticals, Inc.
Approval date: August 10, 2018
on-PAH-troh
Alnylam Pharmaceuticals, Inc.
Approval date: August 10, 2018
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
ONPATTRO is a drug for the treatment of nerve damage in adult patients with hereditary transthyretin-mediated amyloidosis. Transthyretin-mediated amyloidosis is the buildup of abnormal deposits of a substance called amyloid in the body's organs and tissues. Amyloid disrupts the function of organs and tissues.
How is this drug used?
A healthcare provider injects ONPATTRO directly into the bloodstream through a needle in the vein. This is known as an intravenous, or IV infusion. It takes about 80 minutes to receive an ONPATTRO infusion.
ONPATTRO is given once every three weeks.
The amount of drug used depends on the patient’s weight.
What are the benefits of this drug?
Compared to patients receiving placebo infusions, patients who received ONPATTRO had less symptoms of nerve damage, as well as better muscle strength and reflexes.
Were there any differences in how well the drug worked in clinical trials among sex, race and age?
- Sex: ONPATTRO worked similarly in males and females.
- Race: The majority of patients in the trial was White. Differences in how well the drug worked among races could not be determined because of the small number of patients in other races.
- Age: ONPATTRO worked similarly in patients younger and older than 65 years of age.
What are the possible side effects?
ONPATTRO may cause serious side effects, including infusion-related reactions and decreased vitamin A levels.
The most common side effects are upper respiratory tract infections and infusion-site reactions.
Were there any differences in side effects among sex, race and age?
- Sex: The occurrence of side effects was similar in males and females.
- Race: The majority of patients in the trial was White. Differences in the occurrence of side effects among races could not be determined because of the small number of patients in other races.
- Age: The occurrence of side effects was similar in patients younger and older than 65 years of age.
WHO WAS IN THE CLINICAL TRIALS?
Who participated in the clinical trials?
The FDA approved ONPATTRO based on evidence from one clinical trial (Trial 1/NCT01960348) of 225 patients with hereditary transthyretin-mediated amyloidosis. The trial was conducted at 44 sites in Asia, Canada, Central America, Europe, South America, and the United States.
Figure 1 summarizes how many men and women were in the clinical trial used to evaluate efficacy and safety.
Figure 1. Baseline Demographics by Sex
FDA Review
Figure 2 summarizes the percentage of patients by race in the clinical trial used to evaluate efficacy and safety.
Figure 2. Baseline Demographics by Race
FDA Review
Table 1. Demographics of Efficacy Trials by Race
Race
|
Number of Patients
|
Percentage of Patients
|
|---|---|---|
White
|
163
|
73%
|
Black or African American
|
5
|
2%
|
Asian
|
52
|
23%
|
Other
|
5
|
2%
|
FDA Review
Figure 3 summarizes the percentage of patients by age enrolled in the clinical trial used to evaluate efficacy and safety.
Figure 3. Baseline Demographics by Age 
FDA Review
How were the trials designed?
The benefits and side effects of ONPATTRO were evaluated in one clinical trial. The trial enrolled patients who had hereditary transthyretin-mediated amyloidosis. Patients were randomly assigned to receive ONPATTRO or placebo by intravenous infusion, once every 3 weeks for 18 months. All patients received medications to prevent possible allergic reactions from the infusions. Neither the patients nor the health care providers knew which treatment was being given until after the trial was completed.
Health care providers rated the change in the signs and symptoms of neuropathy (nerve damage) from baseline to Month 18 using a numerical scale. The scores for the patients receiving ONPATTRO were compared to the scores for the patients receiving placebo.
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.
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.
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