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 - Seite 2

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 November 2019, Oxbryta has been prescribed to thousands of patients. Analyses from two studies of real-world experience with Oxbryta showed hemoglobin levels increased similarly to what was reported from the HOPE study.

An analysis (Abstract #2627) evaluating Symphony Health claims data from a subset of 1,275 SCD patients ages 12 and older treated with Oxbryta showed statistically significant reductions in annualized transfusion rates and a reduced annual rate of VOC events following the initiation of Oxbryta therapy.

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.