Studied in pediatric (12-17) and adult (>17) patients with sickle cell disease (SCD) in the HOPE trial1
Oxbryta tablets were studied in a multicenter, randomized, double-blind, placebo-controlled (Phase 3) trial of 274 patients aged 12-64 years.
Patients were randomized to either 1,500 mg/day of Oxbryta (n=90), 900 mg/day of Oxbryta tablets (n=92), or placebo (n=92). Randomization was stratified by patients already receiving xydroxyurea (HU), geographic region, and age.
Primary Endpoint1
Percentage of patients who had a hemoglobin (Hb) response (defined as an increase of >1 g/dL) from baseline to Week 24
Secondary Endpoints1,2
Change in Hb level from baseline at Week 24
Change in laboratory markers associated with hemolysis (indirect bilirubin level and percentage of reticulocytes) from baseline at 24 weeks
Patient Details
Select inclusion criteria1
Baseline Hb of 5.5-10.5 g/dL
1-10 vaso-occlusive crisis (VOC) events within the last 12 months
Eligible patients on stable doses of HU for at least 90 days were allowed to continue HU therapy throughout the study
Baseline demographics for patients in the Phase 3 HOPE trial1
The waterfall chart is a visual representation of the data. Each bar represents an individual patient and their Hb change from baseline. The dotted line shows 1 g/dL in the HOPE trial.3
Oxbryta significantly increased Hb levels in the Phase 3 HOPE trial1
51%
of patients (from the intent-to-treat* population) receiving Oxbryta achieved a >1 g/dL increase in Hb (compared to baseline) vs 7% in the placebo group (P<0.001)1
59%
of patients (from the per-protocol† population) receiving Oxbryta achieved a >1 g/dL increase in Hb (compared to baseline) vs 10% in the placebo group (P<0.001)3
Change in Hb for individual patient at Week 241†
Some patients receiving Oxbryta had increases in Hb beyond the primary endpoint3
Increased by >2 g/dL
27%
Oxbryta
vs
1%
Placebo
*Intent-to-treat: Included all randomized patients.3
†Per-protocol: Included randomized patients who completed 24 weeks of study drug and adhered to defined trial protocols (approximately 83% of all randomized patients).1,3
Results from a multicenter, randomized, double-blind, placebo-controlled parallel group study of 274 patients, aged 12-64 years, with SCD (a majority with HbSS or HbSβ0 thalassemia), conducted in 3 groups of study participants over a 24-week period. All data in this section from the per-protocol analysis unless otherwise indicated.1,3
Increases in hemoglobin (Hb) maintained through Week 72 in the HOPE trial4
Mean change in Hb from baseline through Week 72 in the HOPE trial3,4
The change from baseline to Week 24 represents a secondary endpoint and the change from baseline to Week 72 was a prespecified, exploratory endpoint2,4
Selected Safety Information
Warnings and precautions
Hypersensitivity Reactions
Serious hypersensitivity reactions after administration of OXBRYTA have occurred in <1% of patients treated. Clinical manifestations may include generalized rash, urticaria, mild shortness of breath, mild facial swelling, and eosinophilia.
If hypersensitivity reactions occur, discontinue OXBRYTA and administer appropriate medical therapy. Do not reinitiate OXBRYTA in patients who experience these symptoms with previous use.
Laboratory Test Interference
OXBRYTA administration may interfere with measurement of Hb subtypes (HbA, HbS, and HbF) by high-performance liquid chromatography (HPLC). If precise quantitation of Hb species is required, chromatography should be performed when the patient has not received OXBRYTA therapy in the immediately preceding 10 days.
Oxbryta provided a mean incremental increase in Hb from baseline regardless of baseline HU use3
Adjusted mean (95% CI) change in Hb from baseline stratified by baseline HU use
Oxbryta reduced hemolysis in the Phase 3 HOPE trial1
Placebo-adjusted LS mean (95% CI) change from baseline in indirect bilirubin1-4
Results from a multicenter, randomized, double-blind, placebo-controlled, parallel group study of 274 patients, aged 12-65 years, with SCD (a majority with HbSS or HbSβ0 thalassemia), conducted in 3 groups of study participants over a 24-week period (pivotal analysis) and a 72-week period (prespecified end-of-study endpoint).1,4
CI = confidence interval; LS = least squares
Responses are measurable and verifiable in patients with SCD1,3
Oxbryta reduced hemolysis in the Phase 3 HOPE trial1
Placebo-adjusted LS mean (95% CI) change from baseline in percent reticulocyte count1-4
Results from a multicenter, randomized, double-blind, placebo-controlled, parallel group study of 274 patients, aged 12-64 years, with SCD (a majority with HbSS or HbSβ0 thalassemia), conducted in 3 groups of study participants over a 24-week period (pivotal analysis) and a 72-week period (prespecified end-of-study endpoint).1,4
CI = confidence interval; LS = least squares
Responses are measurable and verifiable in patients with SCD1,3
Studied in pediatric patients ages 4 to <12 years with sickle cell disease (SCD) in the Phase 2 HOPE-KIDS 1 trial1,5
Oxbryta was studied in an open-label, multicenter, Phase 2 study of 45 patients.
Patients aged 4 to <12 years received Oxbryta (voxelotor) tablets for oral suspension based on body weight at baseline.
Doses of 600 mg, 900 mg, or 1,500 mg once daily were administered to patients weighing 10 kg to <20 kg, 20 kg to <40 kg, or ≥40 kg, respectively
35 patients received Oxbryta for 24 weeks
26 patients received Oxbryta for 48 weeks
Primary Outcome1,5
Percentage of patients who achieved a response, defined as an Hb increase of >1 g/dL from baseline to Week 24
Secondary Outcome3,5
Percent change in clinical measures of hemolysis from baseline to Week 24
Patient Details
Select inclusion criteria1
Baseline Hb of ≤10.5 g/dL
Eligible patients on stable doses of hydroxyurea ≥90 days were allowed to continue hydroxyurea therapy throughout the study
Select baseline demographics1
Median age was 8 years (range: 4-15 years)
45 (80%) of patients were 4 to <12 years of age
80% received background hydroxyurea therapy
Patients had a mean baseline of 8.6 g/dL (range: 6.1-10.5 g/dL)
All patients had HbSS or HbSβ0 thalassemia genotype (100%)
The waterfall chart is a visual representation of the data. Each bar represents an individual patient and their Hb change from baseline. The dotted line shows 1 g/dL in the HOPE-KIDS 1 trial.3
Oxbryta significantly increased Hb levels in the HOPE-KIDS 1 trial1,3,5
36%
of patients (from the intent-to-treat* population) receiving Oxbryta achieved a response, defined as a Hb increase of >1 g/dL from baseline to Week 24 (95% CI: 21.6%, 49.5%)1,3,5
47%
of patients (from the per-protocol† population) receiving Oxbryta achieved a response, defined as a Hb increase of >1 g/dL from baseline to Week 24 (95% CI: 29.8%, 64.9%)1,3,5
Change in Hb for individual patients 4 to <12 at Week 243
At Week 24 vs baseline
35% of patients receiving Oxbryta achieved >1.5 g/dL increase in Hb from baseline3
21% of patients receiving Oxbryta achieved >2.0 g/dL increase in Hb from baseline3
+1.0 g/dL mean increase in Hb levels above background HU benefits was achieved by those patients receiving Oxbryta who were already on stable HU therapy1,3
Results from an open-label, multicenter, Phase 2 study of 45 patients with SCD. Patients aged 4 to <12 years received Oxbryta tablets for oral suspension based on body weight at baseline. Doses of 600 mg, 900 mg, or 1,500 mg once daily were administered to patients weighing 10 kg to <20 kg, 20 kg to <40 kg, or ≥40 kg, respectively. All data presented in this section from the per-protocol analysis unless otherwise indicated.1,3,5
*Intent-to-treat: Included all patients who enrolled and received at least 1 dose of study drug regardless of adherence to defined trial protocols.3
†Per-protocol: Included patients who completed 24 weeks of study drug and had a Hb measurement available at Week 24.3
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A message from Dr. Ted W. Love
President and CEO, Global Blood Therapeutics, Inc.
Thank you for visiting the virtual 62nd ASH® Annual Meeting and Exposition!
Thank you for visiting the virtual 62nd ASH® Annual Meeting and Exposition!
We are proud to participate in this important meeting and appreciate your interest in learning more about GBT and Oxbryta® (voxelotor) tablets. We at GBT share your passion and commitment to improving the lives of patients. Together, we can make a difference.
I encourage you to explore our website to learn more about Oxbryta.
Some of the uses or data for Oxbryta® (voxelotor) tablets described in the attached journal article have not been approved by the Food & Drug Administration.
NEJM does not hold itself responsible for statements made by any contributor. Statements or opinions expressed in NEJM reflect the views of the author(s) and not the official policy of the Massachusetts Medical Society unless so stated. NEJM reprints are not intended as the sole source of clinical information on this topic. Readers are advised to search the NEJM website at www.nejm.org and other medical sources for relevant clinical information on this topic. Reprints of articles published in NEJM are distributed only as free-standing educational material. They are not intended to endorse or promote any organization or its products or services.
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