A new DRUG TRIALS SNAPSHOT is now available.
OXBRYTA is used for the treatment of sickle cell disease (SCD) in patients 12 years of age and older.
SCD is an inherited blood disorder in which the red blood cells are abnormally shaped (in a crescent or "sickle" shape) because of an abnormal type of hemoglobin (the part of the red blood cell that carries oxygen to all parts of the body). The disease is characterized by hemolytic anemia and recurrent painful vaso-occlusive crises (VOC).
Three OXBRYTA tablets (total of 1500 mg) are taken once daily by mouth.
See more Drug Trials Snapshots or contact us with questions at Snapshots@fda.hhs.gov.
Drug Trials Snapshots: OXBRYTA
OXBRYTA (voxelotor)
ox brye ta
Global Blood Therapeutics
Approval date: November 25, 2019
ox brye ta
Global Blood Therapeutics
Approval date: November 25, 2019
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
OXBRYTA is used for the treatment of sickle cell disease (SCD) in patients 12 years of age and older.
SCD is an inherited blood disorder in which the red blood cells are abnormally shaped (in a crescent or "sickle" shape) because of an abnormal type of hemoglobin (the part of the red blood cell that carries oxygen to all parts of the body). The disease is characterized by hemolytic anemia and recurrent painful vaso-occlusive crises (VOC).
How is this drug used?
Three OXBRYTA tablets (total of 1500 mg) are taken once daily by mouth.
What are the benefits of this drug?
Fifty one percent (46/90) of patients treated with OXBRYTA achieved an increase in hemoglobin level of at least 1g/dl in comparison to 6.5% (6/92) of patients treated with placebo.
OXBRYTA was approved under FDA’s accelerated approval program, which provides earlier patient access to a promising new drug while the company continues to conduct clinical trials to confirm that the drug works well.
More trials are ongoing to assess whether there is a clinical benefit.
Were there any differences in how well the drug worked in clinical trials among sex, race and age?
- Sex: OXBRYTA worked similarly in males and females.
- Race: The majority of patients were Black or African Americans. The number of patients in other races were limited; therefore, differences in how OXBRYTA worked among races could not be determined.
- Age: The majority of patients were adults between 18-64 years of age. There were not enough patients older than 65 years to determine whether there was any difference in how OXBRYTA worked between older and younger patients.
What are the possible side effects?
OXBRYTA may cause serious allergic reactions.
The most common side effects of OXBRYTA include headache, diarrhea, abdominal pain, nausea, fatigue, rash and fever.
Were there any differences in side effects among sex, race and age?
- Sex: The occurrence of side effects was similar between males and females.
- Race: The majority of patients were Black or African Americans. The number of patients in other races were limited; therefore, differences in how OXBRYTA worked among races could not be determined.
- Age: The majority of patients were adults between 18-64 years of age. There were not enough patients older than 65 years to determine whether there was any difference in side effects between older and younger patients.
WHO WAS IN THE CLINICAL TRIALS?
Who participated in the clinical trials?
The FDA approved OXBRYTA based on evidence from one clinical trial (Trial 1/NCT03036813) that enrolled patients with SCD disease. The trial was conducted at 60 sites in the United States, Canada, Egypt, Kenya, Lebanon, Oman, Jamaica, Great Britain, Italy, Netherlands, France and Turkey.
Figure 1 summarizes how many men and women were in the clinical trial.
Figure 1. Baseline Demographics by Sex
Adapted from FDA Review
Figure 2 summarizes the percentage of patients by race in the clinical trial.
Figure 2. Baseline Demographics by Race
* includes White, Arab, Middle Eastern
Adapted from FDA Review
Figure 3 summarizes the percentage of patients by age in the clinical trial.
Figure 3. Baseline Demographics by Age
Adapted from FDA Review
How were the trials designed?
There was one trial that evaluated the benefit and side effects of OXBRYTA in patients with SCD. In this trial, patients were required to have a history of 1-10 VOCs in the previous 12 months and low hemoglobin levels (from 5.5 to 10.5 g/dL). Patients received at random either OXBRYTA or placebo tablets daily for 6 months. Neither the patients nor the healthcare providers knew which treatment was given until the end of the trial.
The benefit of OXBRYTA in comparison to placebo was assessed by a percentage of patients who achieved an increase in Hb level of 1g/dl over a 6 months period.
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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