Oxbryta Sickle Cell Disease Pediatric Indications & Dispersible Tablets Received Priority Review by the U.S. FDA

Global Blood Therapeutics (GBT) recently announced that the U.

Sickle cell disease (SCD) is a devastating disease that can cause organ damage and shortened life expectancy, and it is complicated by the huge difference in access to quality care

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

In the United States, approximately 17,000 children between the ages of 4 and 11 suffer from SCD

NDA seeks approval for 300mg dispersible tablets

In April this year, GBT announced the 72-week data of the Phase 3 HOPE study.

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

Oxbryta can reduce sickling and quickly improve red blood cell health

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: US FDA Accepts for Priority Review Supplemental New Drug Application for Oxbryta (voxelotor) for the Treatment of Sickle Cell Disease in Children Ages 4 to 11