A new DRUG TRIALS SNAPSHOT is now available
Drug Trial Snapshot: TEGSEDI
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 TEGSEDI 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 TEGSEDI Package Insert for complete information.
TEGSEDI (inotersen)
teg-SED-ee
Ionis Pharmaceuticals, Inc.
Approval date:October 5, 2018
teg-SED-ee
Ionis Pharmaceuticals, Inc.
Approval date:October 5, 2018
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
TEGSEDI 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?
TEGSEDI is injected under the skin (a subcutaneous injection) once weekly.
What are the benefits of this drug?
Compared to patients receiving placebo injections, patients who received TEGSEDI had fewer 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: TEGSEDI 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: TEGSEDI worked similarly in patients younger and older than 65 years of age.
What are the possible side effects?
TEGSEDI can cause serious side effects, including low platelet counts and kidney inflammation. Because of these serious side effects, TEGSEDI is available only through a restricted program called the TEGSEDI Risk Evaluation and Mitigation (REMS) Program.
Other serious side effects include stroke, serious inflammatory and immune system problems, abnormal liver function, and decreased vitamin A levels.
The most common side effects are injection site reactions (redness, swelling, bleeding, pain, rash, and itching at the injection site), nausea, headache, tiredness, low platelet counts, and fever.
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 congestive heart failure, muscle and extremity pain, and chills was increased in patients 65 years of age and older.
WHO WAS IN THE STUDIES?
Who participated in the clinical trials?
The FDA approved TEGSEDI based on evidence from one clinical trial (Trial 1/NCT01737398) that included 172 patients with hereditary transthyretin-mediated amyloidosis. The trial was conducted at 24 sites in Australia, Europe, South America, and the United States.
Figure 1 summarizes how many men and women were in the clinical trial.
Figure 1. Baseline Demographics by Sex
FDA Review
Figure 2 summarizes the percentage of patients by race in the clinical trial.
Figure 2. Baseline Demographics by Race
FDA Review
Teble 1. Demographics of Trial by Race
Race
|
Number of Patients
|
Percentage of Patients
|
|---|---|---|
White
|
158
|
93%
|
Black or African American
|
4
|
2%
|
Asian
|
4
|
2%
|
Other
|
5
|
3%
|
FDA Review
Figure 3 summarizes the percentage of patients by age in the clinical trial.
Figure 3. Baseline Demographics by Age
FDA Review
How were the trials designed?
The benefits and side effects of TEGSEDI were evaluated in one clinical trial that enrolled patients with hereditary transthyretin-mediated amyloidosis. Patients were randomly assigned to receive TEGSEDI or placebo by subcutaneous injection given once a week for 65 weeks. During the first week of treatment, patients received 3 doses of treatment, followed by once weekly subcutaneous injections for 64 weeks. 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 Week 66 using a numerical scale. Changes from baseline to Week 66 in the impact of neuropathy on patients’ daily functioning were also evaluated using a numerical scale. The scores for the patients receiving TEGSEDI 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.
.png)
No hay comentarios:
Publicar un comentario