Results With ENDARI
The efficacy and safety of ENDARI in sickle cell disease was evaluated in a clinical study.
The study included 230 patients with sickle cell anemia or sickle β0-thalassemia.
Patients were treated with ENDARI or placebo for 48 weeks.
All enrolled patients had 2 or more painful crises within 12 months prior to enrollment.
ENDARI was shown to lower the frequency of sickle cell crises by 25%
ENDARI: A median number of 3 sickle cell
crises per patient
Placebo: A median number of 4 sickle cell
crises per patient
ENDARI was shown to lower the frequency of hospitalizations by 33%
ENDARI: A median number of 2 hospitalizations for sickle cell pain
Placebo: A median number of 3 hospitalizations for sickle cell pain
ENDARI was shown to reduce cumulative days spent in the hospital by 41%
ENDARI: A median number of 6.5 cumulative days spent in the hospital
Placebo: A median number of 11 cumulative days spent in the hospital
ENDARI was shown to increase time to first sickle cell crisis by 30 days
ENDARI: A median number of 84 days until the first sickle cell crisis
Placebo: A median number of 54 days until the first sickle cell crisis
ENDARI was shown to reduce the occurrence of acute chest syndrome
ENDARI: 13 out of 152 patients (8.6%) experienced acute chest syndrome
Placebo: 18 out of 78 (23.1%) patients experienced acute chest syndrome
| Adverse reaction |
Endari N = 187 (%) |
Placebo N = 111 (%) |
|---|---|---|
| Constipation | 21 | 18 |
| Nausea | 19 | 14 |
| Headache | 18 | 15 |
| Abdominal Pain 1 | 17 | 16 |
| Cough | 16 | 14 |
| Pain in extremity | 13 | 7 |
| Back pain | 12 | 5 |
| Chest pain | 12 | 8 |
| 1 Abdominal pain = abdominal pain and abdominal pain, upper | ||
INDICATION
ENDARI is indicated to reduce the acute complications of sickle cell disease in adults and children 5 years of age and older.
IMPORTANT SAFETY INFORMATION
The most common side effects in clinical studies were constipation, nausea, headache, pain in the stomach area, cough, pain in hands or feet, back pain, and chest pain.
Side effects that lead to a stop in treatment during the clinical study were, one case each of overactive spleen (an organ that helps filter your blood), pain in the stomach area, indigestion, burning sensation, and hot flash.
It is not known whether ENDARI is safe and effective in children with sickle cell disease younger than 5 years old.
Talk to your doctor to determine if prescription ENDARI is right for you. You may report side effects to the FDA. Visit www.fda.gov/MedWatch or call 1-800-FDA-1088. You can also call Emmaus Medical, Inc. at 1-855-725-0900.
For more information, please read the full Prescribing Information, including Instructions for Use, for ENDARI.
