Person living with beta thalassemia and their caregiver sitting on a front porch
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How CASGEVY™ Is Being
Studied

How CASGEVY™ Is
Being Studied

People living with TDT pictured may or may not have received CASGEVY.

Luke, living with TDT,
and his mom, Alison

Luke, living with TDT, and his mom, Alison

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People living with TDT pictured may or may not have received CASGEVY.

The clinical study was designed to determine the possible benefits and risks of CASGEVY.

The clinical study was designed to determine the possible benefits and risks of CASGEVY.

The clinical study was open label, which means everyone in the study knew they were receiving CASGEVY. There was no placebo.

Study Purpose

Information sheet with bar graph and line graph representing the study design and results

Study purpose: The study looked at the safety of CASGEVY and how well CASGEVY worked, like how many people no longer required transfusions for at least 12 months in a row after receiving CASGEVY. The study is ongoing until all patients reach 24 months after receiving CASGEVY.

Participant Details

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Participant details:

  • 52 people in the study received CASGEVY
  • Efficacy, or how well CASGEVY worked, was measured for 35 people at the time data were reviewed
  • Ages 12-35
  • Living with beta thalassemia and need regular blood transfusions. This is also called transfusion-dependent beta thalassemia (or TDT)
  • 52 people in the study received CASGEVY
  • Efficacy, or how well CASGEVY worked, was measured for 35 people at the time data were reviewed
  • Ages 12-35
  • Living with beta thalassemia and need regular blood transfusions. This is also called transfusion-dependent beta thalassemia (or TDT)

Transfusion independence may be possible with CASGEVY

Transfusion independence may be possible with CASGEVY

In the clinical study:

Icon showing 91.4%

of people (32 out of 35 people) did not need a transfusion for at least 12 months in a row after receiving CASGEVY

of people (32 out of 35 people) did not need a transfusion for at least 12 months in a row after receiving CASGEVY

Icon of a calendar showing 20.8 months on average

For the 32 people who did not need a transfusion for at least 12 months in a row:

For the 32 people who did not need a transfusion for at least 12 months in a row:

So far, the average length of time without a transfusion was 20.8 months (measured as a median* amount)

So far, the average length of time without a transfusion was 20.8 months (measured as a median* amount)

*A median amount is the middle value of a group of numbers; half of the numbers are less than the median and half are higher.

Average total hemoglobin and fetal hemoglobin levels over time

Chart showing fetal hemoglobin and adult hemoglobin over time Chart showing fetal hemoglobin and adult hemoglobin over time

Data were not available for every patient at each month. The study results differed among individuals. Your experience may be different.

Data were not available for every patient at each month. The study results differed among individuals. Your experience may be different.

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Start the CASGEVY conversation

Talking with your healthcare provider and loved ones is an important step when considering CASGEVY.

View the discussion guide View the discussion guide
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Safety of CASGEVY

Learn about the safety and side effects of CASGEVY in clinical studies.

See the safety results See the safety results