How May ADAKVEO Help?

How was ADAKVEO studied?

The safety and effectiveness of ADAKVEO were studied in 198 people with sickle cell disease.

67 people received ADAKVEO, and 65 people received placebo at study centers primarily across the United States.

In the 1-year study, pain crises were defined as painful episodes that led to a health care visit (ER, clinic, hospital, or local physician visit). Your doctor or health care provider may refer to pain crises as "vaso-occlusive crises" [vey-soh uh-kloo-siv] or VOCs. The following health conditions were also considered pain crises if they led to a health care visit:

• Respiratory issues, or acute chest syndrome
• Reduced blood flow or cell trapping in the liver, or hepatic sequestration
• Reduced blood flow or cell trapping in the spleen, or splenic sequestration
• Prolonged erections, or priapism

Who received ADAKVEO in the study?

The clinical study of ADAKVEO included people:

Across all genotypes of sickle cell disease, including HbSS, HbSC, HbSß⁰-thalassemia, HbSß⁺-thalassemia, and others

 

Who were of African, Hispanic, Caucasian, and other ancestries

 

Who had either 2-4 or 5-10 painful episodes (crises) in the last 12 months

Who were also on hydroxyurea, and people who were not

What were the results of the ADAKVEO study?

67 people receiving ADAKVEO experienced a median annual rate of 1.63 pain crises compared with 2.98 pain crises in 65 people taking placebo—a 45% reduction. The median is the number that is exactly in the middle of all results seen in the 1-year study.

ADAKVEO reduced the number of pain crises people had regardless of sickle cell disease genotype and whether or not they were taking hydroxyurea.

67 people taking ADAKVEO experienced a median annual rate of 4.0 days in the hospital vs 6.87 days for 65 people taking placebo—a 42% reduction. The median is the number that is exactly in the middle of all results seen in the 1-year study. The annual number of days a patient was hospitalized included those days related to pain crises.

More than one-third of people had no pain crises with ADAKVEO in the clinical study

A total of 24 out of 67 people taking ADAKVEO had no pain crises compared with 11 out of 65 people taking placebo. These results include some people who did not complete the study.