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.
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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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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Now Available: 300 mg tablets
for patients as young as 4 years old1
Oxbryta is available in tablets (300 mg and 500 mg) and tablets for oral suspension (300 mg)
For dosing information, please see Full Prescribing Information