Press Release
GBT Presents New Data on the Long-Term and Real-World Use of Oxbryta® (voxelotor) Tablets in Patients with Sickle Cell Disease at 62nd ASH Annual Meeting and Exposition
Final 72-Week Analyses of Phase 3 HOPE Study Demonstrate Durable Improvements in Hemoglobin Levels and Significant Improvements in Overall Health Status
Real-World Experience Study Results Consistent with HOPE Study and Show Improved Patient Health Status
“We are pleased that the longer term, 72-week HOPE Study data are consistent with the previously reported 24-week primary analyses, confirm the durability of effect and justify the sustained use of Oxbryta for treatment of sickle cell disease,” said
72-Week Analyses of Phase 3 HOPE Study
The analyses of the complete data from the Phase 3 HOPE Study support the long-term use of Oxbryta to reduce anemia and hemolysis, with the potential to mitigate the associated morbidity and mortality of SCD.
An analysis of the 72-week data (Abstract #1716) demonstrated that Oxbryta at 1500 mg resulted in durable improvements in hemoglobin levels and markers of hemolysis over 72 weeks of treatment. A large majority of patients (approximately 90 percent) achieved a Hb improvement of >1 g/dL from baseline at one or more time points during the study as compared to placebo (approximately 25 percent). The study also found:
- Significant improvements in markers of hemolysis in indirect bilirubin and reticulocyte percentage.
- Consistent with the 24-week data previously reported, treatment with Oxbryta remained well tolerated. The most common side effects reported were headache, diarrhea, abdominal pain, nausea, arthralgia, rash and pyrexia.
“The underlying cause of sickle cell disease and the root of the devastating, life threatening complications of the disease is hemoglobin polymerization and the resulting anemia and hemolysis,” said
Another HOPE Study analysis (Abstract #795) found that higher hemoglobin levels achieved with Oxbryta are associated with a lower incidence of vaso-occlusive crises (VOCs) over 72 weeks. While the HOPE Study was not designed or powered to show an effect on VOCs, these results suggest the importance of reducing hemolysis and raising hemoglobin in individuals with SCD through inhibition of polymerization. Specifically:
- The annualized incidence rates of VOCs were numerically lower in patients receiving Oxbryta 1500 mg (2.4) than placebo (2.8); this numerical difference was greater in patients who had experienced two or more VOCs in the year prior to the study.
- Patients with the highest average hemoglobin levels over 72 weeks experienced the fewest VOCs with Oxbryta, with a stepwise reduction in VOC rate as hemoglobin levels increased.
A third analysis from the HOPE Study (Abstract #802) used the Clinical Global Impression of Change (CGI-C) scale, a validated outcomes measure that provides a holistic assessment of the effect of treatment. Results showed that treatment with Oxbryta compared to placebo resulted in a statistically significant higher rating of improved overall patient health status after 72 weeks by the treating physician.
Real-World Experience with Oxbryta
Since its approval in
An analysis (Abstract #2627) evaluating
An additional study (Abstract #1723) from a single-center case series showed that both patients and clinicians observed improved health status based on the Patient Global Impression – Improvement (PGI-I) and the Clinical Global Impression – Improvement (CGI-I) scales to examine patient and clinician perception of health status in patients treated with Oxbryta. In addition, while cases of gastrointestinal side effects were reported at a rate of incidence similar to that as the HOPE Study, patients were successfully managed with adjustments to dosing regimens and persisted on treatment.
“After such an extended period with no new treatments for people with SCD, our hope for Oxbryta was that patients would finally have a therapeutic option that could lessen the daily and often invisible burden of this disease,” said
About Sickle Cell Disease
Sickle cell disease (SCD) affects an estimated 100,000 people in
About Oxbryta® (voxelotor) Tablets
Oxbryta (voxelotor) is an oral, once-daily therapy for patients with sickle cell disease (SCD). Oxbryta works by increasing hemoglobin’s affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT believes Oxbryta blocks polymerization and the resultant sickling and destruction of red blood cells, which are primary pathologies faced by every single person living with SCD. Through addressing hemolytic anemia and improving oxygen delivery throughout the body, GBT believes that Oxbryta has the potential to modify the course of SCD. On
As a condition of accelerated approval, GBT will continue to study Oxbryta in the HOPE-KIDS 2 Study, a post-approval confirmatory study using transcranial Doppler (TCD) flow velocity to assess the ability of the therapy to decrease stroke risk in children 2 to 15 years of age.
In recognition of the critical need for new SCD treatments, the FDA granted Oxbryta Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for the treatment of patients with SCD. Additionally, Oxbryta has been granted Priority Medicines (PRIME) designation from the
GBT plans to seek regulatory approvals to expand the potential use of Oxbryta in
Important Safety Information
Oxbryta should not be taken if the patient has had an allergic reaction to voxelotor or any of the ingredients in Oxbryta. See the end of the patient leaflet for a list of the ingredients in Oxbryta.
Oxbryta can cause serious side effects, including serious allergic reactions. Patients should tell their health care provider or get emergency medical help right away if they get rash, hives, shortness of breath or swelling of the face.
Patients receiving exchange transfusions should talk to their health care provider about possible difficulties with the interpretation of certain blood tests when taking Oxbryta.
The most common side effects of Oxbryta include headache, diarrhea, stomach (abdominal) pain, nausea, tiredness, rash and fever. These are not all the possible side effects of Oxbryta.
Before taking Oxbryta, patients should tell their health care provider about all medical conditions, including if they have liver problems; if they are pregnant or plan to become pregnant as it is not known if Oxbryta can harm an unborn baby; or if they are breastfeeding or plan to breastfeed as it is not known if Oxbryta can pass into breastmilk or if it can harm a baby. Patients should not breastfeed during treatment with Oxbryta and for at least two weeks after the last dose.
Patients should tell their health care provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins and herbal supplements. Some medicines may affect how Oxbryta works. Oxbryta may also affect how other medicines work.
Patients are advised to call their doctor for medical advice about side effects. Side effects can be reported to the FDA at 1-800-FDA-1088. Side effects can also be reported to
Full Prescribing Information for Oxbryta is available at Oxbryta.com.
About
Forward-Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995, including statements containing the words “will,” “anticipates,” “plans,” “believes,” “forecast,” “estimates,” “expects,” and “intends,” or similar expressions. These forward-looking statements are based on GBT’s current expectations and actual results could differ materially. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. GBT intends these forward-looking statements, including statements regarding GBT’s priorities, dedication, focus, goals and vision; safety, efficacy and mechanism of action of Oxbryta and other product characteristics; significance of reducing hemolysis and raising hemoglobin; commercialization, delivery, availability, use, and commercial and medical potential of Oxbryta; inferences drawn from study results and related analyses, including with respect to continued use of Oxbryta; growth of real-world evidence; ongoing and planned studies of Oxbryta and related protocols, activities and expectations; potential expansion of the approved use of Oxbryta for more patients in the
References
Centers for Disease Control and Prevention website. Sickle Cell Disease (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. AccessedJune 3, 2019 .European Medicines Agency . https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125. AccessedJune 12, 2020 .- National Heart, Lung, and
Blood Institute website. Sickle Cell Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease. AccessedAugust 5, 2019 . - Rees DC, et al.
Lancet . 2010;376(9757):2018-2031. - Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.
- Kato GJ, et al. J Clin Invest. 2017;127(3):750-760.
- Caboot JB, et al. Paediatr Respir Rev. 2014;15(1):17-23.
- Oxbryta (voxelotor) tablets prescribing information.
South San Francisco, Calif. Global Blood Therapeutics, Inc. ;November 2019 .
Contact:
GBT
650.410.3258
simmergut@gbt.com
GBT
650.351.7881
croberts@gbt.com
Source: Global Blood Therapeutics, Inc.