TAVALISSE is a drug used to treat adults with low platelet count due to chronic immune thrombocytopenia (ITP) when a prior treatment for ITP has not worked well enough. Chronic immune thrombocytopenia is an autoimmune bleeding disorder. In patients with ITP, the blood doesn't clot as it should because of a low platelet count.
TAVALISSE is a tablet that is taken twice daily. After 4 weeks, the dosage can be increased as necessary, to reduce the risk of bleeding.
Drug Trials Snapshot: Tavalisse
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 TAVALISSE 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 TAVALISSE Package Insert for complete information.
TAVALISSE (fostamatinib disodium hexahydrate)
ta vah leese
Rigel Pharmaceuticals, Inc.
Approval date: April 17, 2018
ta vah leese
Rigel Pharmaceuticals, Inc.
Approval date: April 17, 2018
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
TAVALISSE is a drug used to treat adults with low platelet count due to chronic immune thrombocytopenia (ITP) when a prior treatment for ITP has not worked well enough. Chronic immune thrombocytopenia is an autoimmune bleeding disorder. In patients with ITP, the blood doesn't clot as it should because of a low platelet count.
How is this drug used?
TAVALISSE is a tablet that is taken twice daily. After 4 weeks, the dosage can be increased as necessary, to reduce the risk of bleeding.
What are the benefits of this drug?
More patients treated with TAVALISSE achieved pre-determined platelet counts, in comparison to patients who received placebo.
Were there any differences in how well the drug worked in clinical trials among sex, race and age?
- Sex: TAVALISSE worked similarly in men and women.
- Race: Most of the patients were 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: TAVALISSE worked similarly in patients younger and older than 65 years of age.
What are the possible side effects?
TAVALISSE may cause serious side effects such as high blood pressure (hypertension), increased liver enzymes, decreased white blood cell count (neutropenia), and fetal harm.
The most commonly reported side effects are diarrhea, high blood pressure, nausea, respiratory infection, dizziness, increased liver enzymes, rash, abdominal pain, fatigue, chest pain and decreased white blood cell count.
Were there any differences in side effects among sex, race and age?
- Sex: The occurrence of side effects was similar in men and women.
- Race: Most of the patients in the trials were 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 serious adverse events1 was higher in patients 65 years and older.
1Serious adverse event was defined as any event that resulted in one of the following: death, life-threatening event, required hospitalization or extended a current hospital stay, persistent or significant disability/incapacity, or congenital anomaly or birth defect
WHO WAS IN THE CLINICAL TRIALS?
Who participated in the clinical trials?
The FDA approved TAVALISSE based on evidence from two clinical trials, Trial 1 (NCT02076399) and Trial 2 (NCT02076412) of 150 patients with chronic ITP.
Trial 1 was conducted at 35 sites in the following countries: Australia, Canada, Denmark, Hungary, Italy, Netherlands, United Kingdom, and United States.
Trial 2 was conducted at 23 sites. In the following countries: Austria, Bulgaria, Czech Republic, Germany, Norway, Poland, Romania, and Spain.
Figure 1 summarizes how many men and women were in the clinical trials.
Figure 1. Baseline Demographics by Sex
FDA Review
Figure 2 summarizes the percentage of patients by race in the clinical trials.
Figure 2. Baseline Demographics by Race
Table 1. Baseline Demographics by Race
Race |
Number of Patients
|
Percentage
|
|---|---|---|
White
|
139
|
93%
|
Asian
|
5
|
3%
|
Black or African American
|
4
|
3%
|
Other
|
2
|
1%
|
FDA Review
Figure 3 summarizes the percentage of patients by age in the clinical trials.
Figure 3. Baseline Demographics by Age
FDA Review
How were the trials designed?
The trials enrolled adult patients with immune thrombocytopenic purpura (ITP), whose prior treatment for ITP has not worked well enough. Patients were, however, allowed to continue previous ITP treatment during the trial.
All patients were randomly assigned to receive either TAVALISSE or placebo tablets twice daily for 24 weeks. After 4 weeks, the dose could be increased if necessary to reduce the risk of bleeding.
The benefit of TAVALISSE was assessed based on the percentage of patients who achieved and maintained the pre-determined platelet count between treatment weeks 14 to 24 in TAVALISSE and placebo groups respectively.
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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