CASGEVY® Clinical Trial and Results | CASGEVY® (exagamglogene autotemcel)

How CASGEVY^® Is
Being Studied

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

Kiarra, living with SCD, and her grandparents, Tanique and Robert

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People living with SCD 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 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 went without a severe VOC* for at least 12 months in a row after receiving CASGEVY. This study is ongoing until all patients reach 24 months after receiving CASGEVY.

Participant Details

Participant details:

44 people in the study received CASGEVY
Efficacy, or how well CASGEVY worked, was measured for 31 people at the time data were reviewed
Ages 12-35
Living with sickle cell disease and had at least 2 severe VOCs per year in the 2 years before the clinical study

44 people in the study received CASGEVY
Efficacy, or how well CASGEVY worked, was measured for 31 people at the time data were reviewed
Ages 12-35
Living with sickle cell disease and had at least 2 severe VOCs per year in the 2 years before the clinical study

*In clinical studies, severe VOCs, or vaso-occlusive crises, were defined as a pain event requiring a visit to a medical facility and pain medication or a red blood cell (RBC) transfusion, acute chest syndrome, priapism (persistent and often painful erection of the penis for more than 2 hours), or splenic sequestration (blood trapped in the spleen).

Freedom from severe VOCs may be possible with CASGEVY

In the clinical study:

of people (29 out of 31 people) did not have a severe VOC for at least 12 months in a row after receiving CASGEVY

of people evaluated (30 out of 30 people) were not hospitalized for a severe VOC for 12 months in a row after receiving CASGEVY^†

^†One of the 31 participants was evaluable for severe VOCs but not for hospitalizations.

For the 29 people who did not have a severe VOC for 12 months in a row:

So far, the average length of time without a severe VOC was 22.2 months (measured as a median^‡ amount). One person experienced a severe VOC at month 22.8 and required hospitalization

^‡This was measured as a median amount, which 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