DFDA

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
MODERATE
Number 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
POOR
ICER
$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%

Sources:

researchgate.net
ashpublications.org
fda.gov

WARNING: LIMITED TRIALS AVAILABLE

You can search for trials, but you probably can't join any because the 1% Treaty hasn't passed yet. Most trials are severely limited by lack of funding and bureaucratic barriers. Help change this!

Active Clinical Trials
1 active trial recruiting for Voxelotor in Sickle Cell Disorders

Assessing Physical Function in Sickle Cell Patients Taking Voxelotor

NCT06023199ACTIVE NOT RECRUITINGPHASE2
View Study
6 participants
INTERVENTIONAL
Falls Church, United States
Started: Oct 23, 2023
Completed Clinical Trials
5 completed trials for Voxelotor in Sickle Cell Disorders

Voxelotor Sickle Cell Exercise Study

NCT04581356COMPLETEDPHASE4
View Study
14 participants
INTERVENTIONAL
Fairfax, United States
Started: Sep 21, 2020

Actigraphy Improvement With Voxelotor (ActIVe) Study

NCT04400487COMPLETEDPHASE4
View Study
25 participants
INTERVENTIONAL
Farmington, United States +9 more
Started: Dec 21, 2020

Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)

NCT03036813COMPLETEDPHASE3
View Study
449 participants
INTERVENTIONAL
Birmingham, United States +61 more
Started: Dec 1, 2016

Retrospective Real World Oxbryta® Data Collection and Analysis Study

NCT04930328COMPLETED
View Study
216 participants
OBSERVATIONAL
Farmington, United States +11 more
Started: Mar 20, 2021

Voxelotor CYP and Transporter Cocktail Interaction Study

NCT05981365COMPLETEDPHASE1
View Study
44 participants
INTERVENTIONAL
San Antonio, United States
Started: Apr 17, 2023
Showing 20 of 25 total trials