The clinical safety of Oxbryta in clinical trials

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

  • 48% (42/88) of patients at Week 72 who received Oxbryta required a dose modification (dose reduction or dosing interruption)1,2
    • Most frequent adverse reactions requiring dosage modification occurring in more than 1 patient who received Oxbryta 1,500 mg included diarrhea, headache, rash, nausea, and vomiting2
    • Patients needing a temporary dose modification based on physician discretion or adverse reaction severity, Oxbryta could be lowered to 1,200 mg/day (this was a reduction of 1 pill in the HOPE trial based on the clinical formulation at the time) until the event subsided and patients could return to 1,500 mg/day2
  • During the first 24 weeks, 5% (4/88) permanently discontinued therapy due to a treatment-related adverse reaction, with no additional discontinuations through Week 721,2
  • There were no discontinuations in the Oxbryta 1,500 mg group due to diarrhea, headache, rash, fatigue, arthralgia, or pyrexia2

Adverse reactions (≥10%) in patients receiving Oxbryta (voxelotor) tablets with a difference between arms of >3% compared to placebo at 24 or 72 weeks1,2

Adverse
Reaction*

Duration of treatment

Duration of treatment

Oxbryta

1,500 mg
(n=88)

Placebo

(n=91)

Headache

24 Weeks

26% (23)

22% (20)

72 Weeks

32% (28)

25% (23)

Diarrhea

24 Weeks

20% (18)

10% (9)

72 Weeks

23% (20)

11% (10)

  • Most diarrhea events reported were mild and self-limiting2
    • Median time to onset: 15 days (Q1, Q3: 2, 78)
    • Median time to resolution: 30 days (Q1, Q3: 3, 43)

Abdominal Pain†

24 Weeks

19% (17)

13% (12)

72 Weeks

23% (20)

16% (15)

Nausea

24 Weeks

17% (15)

10% (9)

72 Weeks

19% (17)

10% (9)

Arthralgia

24 Weeks

15% (13)

12% (11)

72 Weeks

22% (19)

14% (13)

Fatigue

24 Weeks

14% (12)

10% (9)

72 Weeks

14% (12)

13% (12)

Rash‡

24 Weeks

14% (12)

10% (9)

72 Weeks

15% (13)

11% (10)

Pyrexia

24 Weeks

12% (11)

7% (6)

72 Weeks

15% (13)

8% (7)

Back pain

24 Weeks

11% (10)

11% (10)

72 Weeks

17% (15)

13% (12)

Additional safety

  • Serious adverse reactions occurred in 3% (3/88) of patients receiving Oxbryta 1,500 mg, which included headache, drug hypersensitivity, and pulmonary embolism occurring in 1 patient each at 24 weeks. No new events occurred after 24 weeks.1,3
  • The safety profile observed in pediatric patients 12 to <17 years of age treated with Oxbryta was similar to that seen in adult patients through Week 24 and Week 721,2
  • Clinically relevant adverse reactions occurring in <10% of patients through Week 24 included drug hypersensitivity. No new events occurred after 24 weeks1,2

*Adverse reactions were Grade 1 or 2, except for Grade 3 diarrhea (n=1), nausea (n=1), rash (n=1), and rash generalized (n=3).

†Abdominal pain (grouped PTs) included the following PTs: abdominal pain and upper abdominal pain.

‡Rash (grouped PTs) includes the following PTs: rash, urticaria, generalized rash, maculo-papular rash, pruritic rash, papular rash, erythematous rash, and vesicular rash.

NEXT: Oxbryta dosing

Clinical safety was studied in the HOPE-KIDS 1 trial1,4

  • 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
  • There were no discontinuations due to diarrhea, vomiting, or rash2
  • 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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