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Primary Endpoint1 | Select Secondary Endpoints1,2 |
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Select inclusion criteria1
Select exclusion criteria1
Baseline demographics for patients in the Phase 3 HOPE trial1
Characteristic | Patients |
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Receiving background HU therapy | 65% |
Geographic region | North America, Europe, other |
Median age | 24 years (12 to 64 years) |
HbSS or HbSβ0 thalassemia genotype | 90% |
Pediatric patients 12 to <17 years | 17% |
Median baseline Hb | 8.5 g/dL (5.9 to 10.8 g/dL) |
1 VOC event in 12 months prior to enrollment | 42% |
2–10 VOC events in 12 months prior to enrollment | 58% |
Responders at 24 weeks
Hb Over 72 weeks (prespecified exploratory)
51% of patients (from the intent-to-treat* population) receiving Oxbryta had a
>1 g/dL increase in Hb (compared to baseline) vs 7% receiving placebo (P<0.001).
Per-protocol analyses provide an estimate of efficacy but may not represent the circumstances seen in clinical practice, as these analyses are limited by reduced sample size and bias due to potential differential exclusion. Results are presented for descriptive purposes and offer supportive, but not conclusive, information.
59% of patients (from the per-protocol† population) receiving Oxbryta had a >1 g/dL increase in Hb (compared to baseline); 10% of patients receiving placebo2,3
Post hoc analyses at week 24 in the per-protocol population3,†:
The results beyond week 24 are from a prespecified exploratory analysis and are not included in the Oxbryta USPI. Results are presented for descriptive purposes and offer supportive, but not conclusive, information.
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
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.
Drug reaction with eosinophilia and systemic symptoms (DRESS) has been reported in postmarketing experience with Oxbryta. Patients who develop a combination of skin rash, fever, peripheral eosinophilia, and internal systemic organ involvement (e.g., hepatic, renal, pulmonary) while receiving Oxbryta should undergo medical evaluation.
Advise patients of the signs and symptoms of severe hypersensitivity reactions, including DRESS. 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.
These results are from an exploratory analysis and are not included in the Oxbryta USPI. Subgroups were prespecified but not alpha protected (controlled for type 1 error), and hierarchical testing was not conducted. Results are presented for descriptive purposes and offer supportive, but not conclusive, information.
Adjusted mean change in Hb from baseline by background HU use
Indirect Bilirubin
Percent Reticulocytes
LS mean change from baseline in indirect bilirubin at week 24 (secondary endpoint)1,3
LS mean change from baseline in indirect bilirubin at week 723,4
The results at week 72 are from a prespecified exploratory analysis and are not included in the Oxbryta USPI. Results are presented for descriptive purposes and offer supportive, but not conclusive, information.
Results from a multicenter, randomized, double-blind, placebo-controlled, parallel group study of 274 patients, aged 12-64, with SCD (a majority with HbSS or HbSß0 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).1,2,4
LS = least squares.
LS mean change from baseline in percent reticulocyte count at week 723,4
The results at week 72 are from a prespecified exploratory analysis and are not included in the Oxbryta USPI. Results are presented for descriptive purposes and offer supportive, but not conclusive, information.
Results from a multicenter, randomized, double-blind, placebo-controlled, parallel group study of 274 patients, aged 12-64, 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).1,2,4
LS = least squares.
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Oxbryta is indicated for the treatment of sickle cell disease (SCD) in adults and pediatric patients 4 years of age and older.
This indication is approved under accelerated approval based on increase in hemoglobin (Hb). Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).