U.S. flag An official website of the United States government

On Oct. 1, 2024, the FDA began implementing a reorganization impacting many parts of the agency. We are in the process of updating FDA.gov content to reflect these changes.

  1. Home
  2. Drugs
  3. Development & Approval Process | Drugs
  4. Drug Approvals and Databases
  5. Drug Trials Snapshots: REZLIDHIA
  1. Drug Approvals and Databases

Drug Trials Snapshots: REZLIDHIA

HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the key clinical trials that supported the original FDA approval of this drug, and whether there were differences among sex, race, age, and ethnic 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 healthcare provider about the benefits and risks of a drug.

Some of the information in this Snapshot is for presentation purposes and does not represent the approved conditions of use of this drug. Refer to the REZLIDHIA Prescribing Information for all of the approved conditions of use of this drug (e.g., indication(s), population(s), dosing regimen(s), safety information).

Snapshots are limited to the information available at the time of the original approval of the drug and do not provide information on who participated in clinical trials that supported later approvals for additional uses of the drug (if applicable).

REZLIDHIA (olutasidenib)
REZ-LID-EE-AH
Rigel Pharmaceuticals, Inc
Original Approval date
: 12/1/2022


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

REZLIDHIA is a prescription medicine used to treat adults with acute myeloid leukemia (AML) with an isocitrate dehydrogenase-1 (IDH1) mutation when the disease has come back (relapsed) or has not improved after one or more treatments (refractory). Your healthcare provider will perform a test to make sure that REZLIDHIA is right for you.

How is this drug used?

The recommended dose of REZLIDHIA is 150 mg taken by mouth twice daily (about 12 hours apart) around the same times each day. REZLIDHIA should be taken on an empty stomach at least 1 hour before or 2 hours after a meal.

Who participated in the clinical trials?

The FDA approved REZLIDHIA based on evidence from one clinical trial (NCT02719574) of 153 adult patients with AML whose disease has come back or has not improved with previous treatments. A total of 147 patients had a certain type of mutation called IDH1, which was confirmed using an FDA-approved test. The trial was conducted in the United States, Australia, Canada, France, Germany, Italy, Republic of Korea, Spain, and the United Kingdom.

How were the trials designed?

There was one trial that evaluated the benefit and side effects of REZLIDHIA in adult patients with AML whose disease has come or has not improved after previous treatments. All patients had a certain type of mutation called IDH1, which was confirmed with an FDA-approved test.

Patients received REZLIDHIA 150 mg by mouth twice daily. Treatment continued until either disease worsened or patients experienced unacceptable side effects.

The benefit of REZLIDHIA was evaluated by measuring how many patients had a 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.

DEMOGRAPHICS SNAPSHOT

Figure 2. Demographics by Sex

Pie chart summarizing how many male and female patients were in the clinical trial. In total, 79 (52%) male patients and 74 (48%) female patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 3. Demographics by Race

Pie chart summarizing how many White, Black or African American, Asian, and other patients were in the clinical trial. In total, 71 (46%) White patients, 5 (3%) Black or African American patients, 5 (3%) Asian patients, 16 (11%) Other patients, and 56 (37%) patients where race was not provided participated in the clinical trial.

Source: Adapted from FDA Review
* Collection of race data not allowed in certain European nations per country specific regulations.

Figure 4. Baseline Demographics by Age

Pie chart summarizing how many patients by age were in the clinical trial. In total, 37 (24%) patients younger than 65 years of age, 68 (45%) patients between 65 and 75 years of age, and 48 (31%) patients 75 years of age and older participated in the clinical trial.

Source: Adapted from FDA Review

What are the benefits of this drug?

In a clinical trial with 147 adult patients with relapsed or refractory AML with an IDH1 mutation, 35% of patients had 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. The time to this response was about 2 months, and the response lasted about 26 months.

Of the 86 patients who needed blood and/or platelet transfusions before treatment with REZLIDHIA, 34% no longer needed transfusions for at least 56 days during treatment.

Of the 61 patients who did not need transfusions of both blood and platelets before treatment with REZLIDHIA, 64% went at least 56 days without needing a transfusion during treatment.

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

  • Sex: REZLIDHIA worked similarly in males and females.
  • Race: The number of patients of races other than White was small; therefore, differences in how well the drug worked among races could not be determined.
  • Age: REZLIDHIA worked similarly in patients younger and older than 65 years of age.

What are the possible side effects?

REZLIDHIA may cause a serious and life-threatening side effect called differentiation syndrome. Another serious side effect is liver toxicity.

The most common side effects of REZLIDHIA are abnormal liver function tests, nausea, feeling tired or unwell, joint pain, constipation, shortness of breath, fever, rash, mouth sores, diarrhea, and changes in certain blood tests.

Were there any differences in side effects of the clinical trials among sex, race, and age?

  • Sex: The occurrence of side effects was similar in males and females.
  • Race: The number of patients with known races other than White was small; therefore, meaningful differences in the occurrence of side effects among races could not be determined.
  • Age: The occurrence of side effects was similar in patients younger and older than 65 years of age.

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 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.

LINK TO DRUG PACKAGE INSERT

Back to Drug Trials Snapshots

Back to Top