Drug Trials Snapshots: TIBSOVO

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.

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 TIBSOVO Prescribing Information for complete information.

TIBSOVO (ivosidenib)
tib-SOH-voh
Agios Pharmaceuticals
Approval date: July 20, 2018

DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

TIBSOVO is used to treat adults with acute myeloid leukemia (AML) that have a mutation in a gene called IDH1 and whose disease has come back or has not improved after previous treatment(s).

AML is a rapidly progressing cancer that forms in the bone marrow and can result in an increased number of white blood cells in the bloodstream.

How is this drug used?

TIBSOVO is a tablet. Two tablets (total of 500 mg) are taken once a day.

What are the benefits of this drug?

Of the 174 patients who received TIBSOVO, 33 percent experienced no evidence of disease and full recovery of blood counts (complete remission) or no evidence of disease and partial recovery of blood counts after treatment, which lasted about 8 months.

Of the 110 patients who required transfusions of blood or platelets due to AML at the start of the trial, 37 percent went at least 56 days without requiring a transfusion after treatment with TIBSOVO.

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Were there any differences in how well the drug worked in clinical trials among sex, race and age?

Sex: TIBSOVO 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: TIBSOVO worked similarly in patients younger and older than 65 years of age.

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What are the possible side effects?

TIBSOVO may cause a serious and life threating side effect called differentiation syndrome. Other serious side effects include changes in the electrical activity of heart (QTc prolongation) and a nervous system problem called Guillain-Barré syndrome.

Common side effects of TIBSOVO are fatigue, increased white cell blood count, joint pain, diarrhea, shortness of breath, body swelling and nausea.

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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 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 side effects was similar in patients younger and older than 65 years of age.

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WHO WAS IN THE CLINICAL TRIALS?

Who participated in the clinical trials?

The FDA approved TIBSOVO based on evidence from one clinical trial (NCT02074839) of 179 patients with AML whose disease has come back or has not improved after previous treatment(s). A total of 174 patients had a certain type of mutation (IDH1-mutation) which was confirmed using an FDA-approved test.

The trial was conducted in United States and France.

Figure 1 summarizes how many men and women were in the clinical trial.

Figure 1. Baseline Demographics by Sex

FDA review

Figure 2 and Table 1 below summarize the percentage of patients by race in the clinical trial.

Figure 2. Baseline Demographics by Race

FDA review

Table 1. Baseline Demographics by Race

Race	Number of Patients	Percentage
White	112	63
Black or African American	10	6
Asian	6	3
Other	9	5
Not reported or Unknown*	42	23

* collection of data not allowed by authority

FDA review

Figure 3 summarizes how many patients of a certain age were enrolled in the clinical trial.

Figure 3. Baseline Demographics by Age

FDA review

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How were the trials designed?

There was one trial that evaluated the benefit and side effects of TIBSOVO in patients with AML whose disease has come back or has not improved after previous treatment(s). All patients had a certain type of mutation (IDH1-mutation) which was confirmed using an FDA-approved test.

Patients received TIBSOVO once a day until disease worsened or unacceptable toxicity.

The benefit of TIBSOVO was evaluated by measuring:

how many patients reached complete remission (no evidence of disease) with full or partial recovery of blood counts after treatment,
how long those patients remained in remission,
and the percentage of patients who no longer required transfusions after treatment.

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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.