Global Blood Therapeutics’ Oxbryta Greenlit for Pediatric SCD Patients Ages 4 to 11

Shares of Global Blood Therapeutics were up more than 10% in late-afternoon trading following regulatory approval of a supplemental New Drug Application for oral Oxbryta (voxelotor) tablets for the treatment of pediatric sickle cell disease (SCD).

The U.S. Food and Drug Administration greenlit Oxbryta under accelerated approval for the treatment of sickle cell disease in patients ages four to less than 12 years old. The approval expands the previous approval of Oxbryta to treat SCD in patients ages 12 years and older in the United States. Oxbryta is the first FDA-approved treatment for children with SCD that directly inhibits sickle hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD.

SCD affects more than 100,000 people in the United States and millions of people across the globe. In the United States, there are an estimated 16,000 children between the ages of 4 to 11 years who have SCD. The disease, caused by a genetic mutation, causes the production of abnormal hemoglobin, which is known as sickle hemoglobin. The red blood cells become sickle-shaped and can cause blockages in blood vessels, which can lead to life-threatening complications, including stroke and irreversible organ damage.

Ted W. Love, president and chief executive officer of Global Blood Therapeutics, expressed his excitement about the latest approval of Oxbryta. Love said the sickle cell disease community has been “profoundly underserved” for many decades. Because SCD can cause irreversible organ damage, early intervention is of critical importance, he said.

Complications of SCD that begin in early childhood can include neurocognitive impairment, acute chest syndrome, and overt stroke, among other serious issues, he said. Additionally, SCD has been known to limit children’s educational attainment and social lives due to disease complications that typically lead to repeated hospitalizations.

Today’s FDA approval of Oxbryta for children as young as 4 years old – and with a pediatric-friendly dosage form – is an important advance in the treatment of this devastating, lifelong condition. GBT is proud to lead the development of new medicines to address the inadequacies of care for SCD patients,” Love said in a statement.

With the latest approval, Oxbryta will be available in two dosage forms for patients 4 years and older based on the patient’s age, weight, and ability to swallow tablets. One is a traditional tablet, while the other is a dispersible form that can be dissolved into water.

The latest approval was based on data from the open-label Phase 2a HOPE-KIDS 1 Study. In that study, the data showed that weight-based treatment with the dispersible tablet formulation of Oxbryta resulted in rapid and sustained improvements in hemoglobin. Also, the study showed that the drug led to concurrent reduction of hemolysis, or red blood cell destruction.

As a condition of accelerated approval, GBT will continue to examine Oxbryta in the HOPE-KIDS 2 Study, which is a post-approval confirmatory study. GBT will continuing to conduct the HOPE-KIDS 1 Study to demonstrate the benefit of Oxbryta and support potential use in children as young as 9 months of age who have SCD. The company is also conducting an ongoing open-label extension study for all patients less than 18 years of age who have participated in Oxbryta clinical trials.