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About Oxbryta®Clinical Trial DataSafety ProfileDosing Experience with Oxbryta Supporting ResourcesSupporting ResourcesContact UsVideosDownloadable resources

Click here for Oxbryta®▼ (voxelotor) Prescribing Information. Adverse event reporting information can be found at the bottom of the page.

Clinical trial data Clinical paper download

Explore key clinical papers and a trial data summary below

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The efficacy and safety profile of Oxbryta® (voxelotor) were evaluated in the Haemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerisation (HOPE) global clinical trial1
 

HOPE: study design1,2

Patients receiving a stable dose of hydroxycarbamide for ≥3 months were eligible for inclusion1

*A hierarchical testing procedure was implemented in the following order until the p -value exceeded 0.0481: percentage of participants who had a haemoglobin (Hb) response, absolute change in Hb level, relative change in indirect bilirubin level, relative change in absolute reticulocyte count and % reticulocytes, and relative change in LDH level.1
Defined as acute painful crisis or acute chest syndrome for which there was no explanation other than vaso-occlusive crisis and that met the protocol-specified criteria.1
In addition, patients were permitted to continue on a background of standard of care treatment.
HOPE: inclusion and exclusion criteria1,3,4

Key inclusion/exclusion criteria are noted here; the lists are not exhaustive.
 

HOPE included a broad group of patients with a range of clinical characteristics1 Significantly more patients treated with Oxbryta® vs placebo achieved a haemoglobin response at 24 weeks (primary endpoint)1Primary endpoint: percentage of patients treated with Oxbryta® 1,500 mg once daily or placebo who had an Hb response (defined as an Hb increase of >1.0 g/dL [0.62 mmol/L] from baseline at Week 24; ITT analysis)1 Oxbryta® was associated with rapid improvements in haemoglobin levels (secondary endpoint)1,3
  • Secondary endpoint: change in Hb level from baseline* to Week 24 (ITT analysis).1 In the secondary analyses the type I error rate was not protected and therefore the significance (p-value) is not robust
  • Improvements in Hb level with Oxbryta® 1,500 mg/day were observed from as early as 2 weeks1,3

Improvements in Hb levels from baseline to Week 24 were observed in most patients receiving Oxbryta® (secondary endpoint)1,3

  • Secondary endpoint: change in Hb level from baseline to Week 241
  • Shown here are individual patient-level changes in Hb from baseline to Week 24 among patients who completed 24 weeks of treatment (per protocol analysis)3
Reductions in some markers of haemolysis were observed in patients receiving Oxbryta® (secondary and exploratory endpoints)1,3
  • Secondary endpoint: change from baseline to Week 24 in laboratory markers associated with haemolysis1
  • Pre-specified exploratory endpoint: change from baseline to Week 72 in laboratory markers associated with haemolysis7
Trends were observed in the Hb response rate favouring Oxbryta® vs placebo in most subgroups evaluated (pre-specified subgroup analysis)4,5,6
  • Shown here are differences in the percentages of patients treated with Oxbryta® 1,500 mg once daily or placebo who had an Hb response (defined as an Hb increase of >1.0 g/dL [0.62 mmol/L] from baseline at Week 24) across subgroups4
Improvements in haemoglobin levels with Oxbryta® were maintained with long-term follow-up (exploratory endpoint)3,7
  • The increase in Hb levels from baseline with Oxbryta® 1,500 mg/day was maintained through Week 72 in the HOPE trial3,7
  • Pre-specified exploratory endpoint: change in Hb levels from baseline to Week 727
Adverse events reported in HOPE: long-term follow-up7
  • The median duration of follow-up was 72.1 weeks (IQR 42.3–73.0) in the Oxbryta® group and 72.1 weeks (IQR 61.2–72.9) in the placebo group7
  • The most frequently reported adverse events unrelated to sickle cell disease with an incidence of ≥20% in the Oxbryta® group were headache, diarrhoea and arthralgia7
  • Serious adverse events unrelated to sickle cell disease were generally balanced across the treatment groups (25/88 [28%] patients in the Oxbryta® group and 23/91 [25%] patients in the placebo group)7

ACS, acute chest syndrome; CI, confidence interval; Hb, haemoglobin; HbSS, haemoglobin SS disease; IQR, interquartile range; ITT, intention-to-treat; LDH, lactate dehydrogenase; LS, least squares; PP, per protocol; RBC, red blood cell; SE, standard error; VOC, vaso-occlusive crisis.

Clinical paper download
References:
1. Vichinsky E, et al. N Engl J Med. 2019;381(6):509–519
2. ClinicalTrials.gov Identifier: NCT03573882. Available at https://clinicaltrials.gov/ct2/show/NCT03573882. Accessed August  2023
3. Oxbryta® (voxelotor) Summary of Product Characteristics
4. Vichinsky E, et al. N Engl J Med. 2019;381(6):509–519. Supplementary information
5. Oxbryta® EPAR Assessment Report. Available at https://www.ema.europa.eu/en/medicines/human/EPAR/oxbryta. Accessed August 2023
6. Trial Protocol_Vichinsky E, et al. A phase 3 randomized trial of voxelotor in sickle cell disease. N Engl J Med 2019;381:509-19
7. Howard J, et al. Lancet Haematol. 2021;8(5):e323–e333
8. Glaros AK, et al. Ther Adv Hematol. 2021;12:20406207211001136
9. Herity LB, et al. Ann Pharmacother. 2021;55(2):240–245
HOPE study About Oxbryta

An introduction to Oxbryta® – a first-in-class haemoglobin S polymerisation inhibitor3,8,9

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Mode of action

Watch a video demonstrating the mechanism of action of Oxbryta®

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Adverse events should also be reported to Pfizer Medical Information on 01304 616161

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