Oxbryta, the first treatment for sickle cell disease, announces latest research results

Global Blood Therapeutics (GBT) recently evaluated Oxbryta (voxelotor) for the treatment of hemolytic anemia in patients with sickle cell disease (SCD).

Oxbryta is a first-in-class, once-a-day, oral drug that directly inhibits hemoglobin polymerization, which is the root cause of sickling and destruction of SCD red blood cells.

Sickle cell disease (SCD) is a serious, progressive, and debilitating genetic disease that results in abnormal production of sickle hemoglobin (HbS) due to mutations in the β-globin gene.

Oxbryta is the first drug approved to directly inhibit sickle hemoglobin polymerization to treat SCD.

Oxbryta's active drug is called voxelotor (formerly GBT440), which works by increasing the affinity of hemoglobin for oxygen.

Oxbryta was approved by the U.

In January of this year, the European Medicines Agency (EMA) accepted Oxbryta’s marketing authorization application (MAA), which seeks EMA to grant Oxbryta full approval for the treatment of hemolytic anemia in sickle cell disease (SCD) patients 12 years and older .

HOPE is a randomized, double-blind, placebo-controlled, international, multi-center phase 3 study that studied 274 SCD patients aged 12 to 65 years.

The specific data are: about 90% of the patients in the Oxbryta treatment group at the 1500 mg dose improved their hemoglobin level> 1g/dL at one or more time points during the study period, while the proportion of patients in the placebo group who achieved this goal was only 25 %.

In addition, approximately 74% of the patients in the Oxbryta treatment group (n=39/53) were rated by clinicians as "moderately improved" or "very improved", compared with 47% in the placebo group (n=24/51).

The post-hoc analysis of the HOPE study recently published in the American Journal of Hematology assessed the incidence and prognosis of leg ulcers in SCD patients.

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Original Source: Complete 72-Week Results from Phase 3 HOPE Study of Oxbryta (voxelotor) Tablets Published in The Lancet Haematology Show Significantly Improved Hemoglobin, Hemolysis and Overall Health Status in Sickle Cell Disease Patients

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