The clinical safety of Oxbryta in clinical trials

Adverse reactions in adults and adolescents in the HOPE trial through week 72

Adverse reactions (≥10%) in patients receiving Oxbryta (voxelotor) tablets with a difference between arms of >3% compared to placebo through week 721,2

Adverse
Reaction*

Oxbryta

1,500 mg
(n=88)

Placebo

(n=91)

Headache

32% (28)

25% (23)

Abdominal Pain

23% (20)

16% (15)

Nausea

19% (17)

10% (9)

Rash

15% (13)

11% (10)

Pyrexia

15% (13)

8% (7)

*Adverse reactions were Grades 1 or 2 except for Grade 3 headache (2), diarrhea (1), nausea (1), rash (1), and rash generalized (3).
Included abdominal pain, lower abdominal pain, and upper abdominal pain.
Included rash, urticaria, generalized rash, macular rash, maculo-papular rash, pruritic rash and papular rash.

Safety considerations

  • In the HOPE trial, dose modifications (dose reduction or dosing interruption) due to a non–treatment related adverse event occurred in 48% (42/88) of patients who received Oxbryta 1,500 mg2
    • Dose modifications (dose reduction or dosing interruption) due to an adverse reaction occurred in 13.6% (12/88) of patients who received Oxbryta 1,500 mg1
    • The adverse reactions requiring dose modification included rash (4.5%), diarrhea (3.4%), headache (2.3%), nausea (2.3%), abdominal pain (1.1%), and drug hypersensitivity (1.1%)1
  • The safety profile observed through week 24 was similar to that observed through week 721,2
  • Serious adverse reactions occurred in 3% (3/88) of patients who received Oxbryta 1,500 mg, which included headache, drug hypersensitivity, and pulmonary embolism occurring in 1 patient each1
  • The safety profile observed in pediatric patients 12 to <17 years of age treated with Oxbryta was similar to that seen in adult patients1
  • Clinically relevant adverse reactions occurring in <10% of patients who received Oxbryta 1,500 mg included drug hypersensitivity1
  • Permanent discontinuation due to an adverse reaction (Grades 1-4) occurred in 5% (4/88) of patients who received Oxbryta 1,500 mg1

Clinical safety was studied in the HOPE-KIDS 1 trial1,2

  • The safety of Oxbryta in pediatric patients 4 to <12 years with SCD was evaluated in an open-label, Phase 2 study1
    • 45 patients 4 to <12 years of age received doses of Oxbryta tablets for oral suspension based on weight at baseline1
      • 35 patients received Oxbryta for 24 weeks1
      • 26 patients received Oxbryta for 48 weeks1
  • The most common adverse reactions (>10%) reported in pediatric patients 4 to <12 years were pyrexia (36%), vomiting (33%), rash (20%), abdominal pain (18%), diarrhea (18%), and headache (18%)1
  • The overall safety profile of Oxbryta in pediatric patients 4 to <12 years was similar to that seen in adults and pediatric patients ≥12 years1

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