The Drug Trials Snapshots for RYDAPT are now available. RYDAPT was approved for the treatment of both advanced systemic mastocytosis (SM) and acute myeloid leukemia (AML) that has a mutation in a gene called FLT3, in combination with chemotherapy.
Separate Snapshots are available for each indication: RYDAPT for advanced systemic mastocytosis (SM) and RYDAPT for acute myeloid leukemia (AML) that has a mutation in a gene called FLT3, in combination with chemotherapy.
See more Drug Trials Snapshots or contact us with questions at Snapshots@fda.hhs.gov.
Drug Trials Snapshot: RYDAPT
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 RYDAPT Prescribing Information 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 RYDAPT Prescribing Information for complete information.
RYDAPT (midostaurin)
rye-dapt
Novartis Pharmaceuticals Corp.
Approval date: April 28, 2017
rye-dapt
Novartis Pharmaceuticals Corp.
Approval date: April 28, 2017
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
RYDAPT is used to treat adults with advanced systemic mastocytosis (SM). It is to be used in patients who have:
- aggressive SM,
- SM with associated blood cancer, or
- mast cell leukemia.
Advanced mastocytosis (SM) are group of rare and fatal disorders in which the body produces too many mast cells, a type of white blood cell.
How is this drug used?
RYDAPT is a capsule. Four capsules (a total of 100 mg) are taken two times a day with food.
What are the benefits of this drug?
After 6 cycles of treatment with RYDAPT, twenty one percent of 89 patients reached either complete and incomplete remission.
Were there any differences in how well the drug worked in clinical trials among sex, race and age?
- Sex: RYDAPT worked similarly in men and women.
- Race: The majority of participants in the clinical trials were White. Differences among races could not be determined due to small number of participants in other races.
- Age: RYDAPT worked similarly in patients younger and older than 65 years of age.
What are the possible side effects?
RYDAPT can cause serious lung toxicity and harm to an unborn baby.
Common side effects of RYDAPT are nausea, vomiting, diarrhea, body swelling, musculoskeletal pain, abdominal pain, tiredness, upper respiratory tract infection, constipation, fever, headache, and shortness of breath.
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: The majority of patients in the clinical trials were White. Differences in side effects among races could not be determined due to 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 RYDAPT based on evidence from two clinical trials of 142 patients total with advanced mastocytosis. The trials were conducted in 31 centers in Europe, North America, and Australia.
Presented below are patients from both trials who took at least one dose of RYDAPT. This is called the safety population.
Figure 1 summarizes how many men and women were in the clinical trials.
Figure 1. Baseline Demographics by Sex
Clinical trial data
Figure 2 and Table 1 below summarize the percentage of patients by race in the clinical trials.
Figure 2. Baseline Demographics by Race
Clinical trial data
Table 1. Baseline Demographics by Race
Race | Number of Patients | Percentage |
|---|---|---|
White | 132 | 93 |
Black or African American | 2 | 1 |
Unknown | 8 | 6 |
Clinical trial data
Figure 3 summarizes how many patients of a certain age were enrolled in the clinical trials.
Figure 3. Baseline Demographics by Age
Clinical trial data
How were the trials designed?
There were two trials that evaluated benefit and side effects of RYDAPT. All patients had advanced mastocytosis and received RYDAPT two times a day until progression of the disease or unacceptable side effects.
In Trial 1, the benefit of RYDAPT was evaluated after 6 cycles of therapy by measuring how well the patients responded and by how long that response lasted. In Trial 2, the benefit of RYDAPT was evaluated after 2 cycles of therapy by measuring how well the patients responded and by how long that response lasted.
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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