Voxelotor
Treatment for Sickle Cell Disorders
Typical Dosage: 1500 mg daily
Effectiveness
68%
Safety Score
70%
Clinical Trials
25
Participants
1.5K
Comparative Safety Scale(Higher is safer)
Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
70
DangerousModerateSafe
Treatment Details
Dosage Range
1500 mg daily
Time to Effect
1-2 weeks for Hb, 3-6 months for VOCs
Treatment Duration
Lifetime
Evidence Quality
MODERATENumber Needed to Treat (NNT)
7(Treat 7 patients to see 1 additional successful outcome)
Confidence Score
90%confidence in effectiveness data
Health Economics
Annual Cost of Care
Drug Cost:$125,000
Monitoring:$500
Side Effect Mgmt:$200
Total Annual:$125,700
Cost-Effectiveness Analysis
Cost-Effectiveness Rating
POORICER
$750,000/QALY
QALYs Gained
0.18
Outcome-Based Costs
Cost per Responder
$359,143
Comparison vs Hydroxyurea (and standard supportive care)
Cost Difference
+$125,000/year
More expensive
QALY Difference
+0.18 QALYs
Better outcomes
Dominance
No dominance
Voxelotor Outcomes
for Sickle Cell Disorders
Efficacy Outcomes
Overall Effectiveness
+68%
Response Rate
+35%
Common Side Effects
Headache
+25%
Diarrhea
+20%
Abdominal pain
+15%
Nausea
+10%
Active Clinical Trials
No active trials currently recruiting for this treatment
No active trials found in ClinicalTrials.gov
Completed Clinical Trials
6 completed trials for Voxelotor in Sickle Cell Disorders
Voxelotor Sickle Cell Exercise Study
NCT04581356COMPLETEDPHASE4
14 participants
INTERVENTIONAL
Fairfax, United States
Started: Sep 21, 2020
Actigraphy Improvement With Voxelotor (ActIVe) Study
NCT04400487COMPLETEDPHASE4
25 participants
INTERVENTIONAL
Farmington, United States +9 more
Started: Dec 21, 2020
Assessing Physical Function in Sickle Cell Patients Taking Voxelotor
NCT06023199COMPLETEDPHASE2
6 participants
INTERVENTIONAL
Falls Church, United States
Started: Oct 23, 2023
Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
NCT03036813COMPLETEDPHASE3
449 participants
INTERVENTIONAL
Birmingham, United States +61 more
Started: Dec 1, 2016
Retrospective Real World Oxbryta® Data Collection and Analysis Study
NCT04930328COMPLETED
216 participants
OBSERVATIONAL
Farmington, United States +11 more
Started: Mar 20, 2021
Voxelotor CYP and Transporter Cocktail Interaction Study
NCT05981365COMPLETEDPHASE1
44 participants
INTERVENTIONAL
San Antonio, United States
Started: Apr 17, 2023
Showing 20 of 25 total trials