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

Shares of Global Blood Therapy rose more than 10% in late afternoon trading following regulatory approval of an additional new drug application for oral Oxbryta (voxelotor) tablets to treat sickle cell disease in children.

The US Food and Drug Administration has given Oxbryta the green light under accelerated approval for the treatment of sickle cell disease in patients aged four to less than 12 years. The approval extends the previous approval of Oxbryta for the treatment of SCD in patients 12 years of age and older in the United States. Oxbryta is the first FDA-approved treatment for children with SCZ that directly inhibits the polymerization of sickle hemoglobin, the main cause of the sickle formation and destruction of red blood cells in SCZ.

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

Ted W. Love, president and chief executive officer of Global Blood Therapeutics, expressed excitement at Oxbryta’s latest approval. Love said the sickle cell disease community has been “deeply undervalued” for decades. Because SCD can cause irreversible organ damage, early intervention is critical, he said.

Complications of SCD that start in early childhood can include neurocognitive impairment, acute chest syndrome and overt stroke, he said. In addition, SCD is known to limit children’s educational attainment and social life due to disease complications that typically lead to repeated hospitalizations.

Oxbryta’s FDA approval for children as young as 4 years old – and in a pediatric-friendly dosage form – is a major advance in the treatment of this devastating, lifelong condition. GBT is proud to lead the development of new drugs to address the shortcomings 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 in 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 Oxbryta’s dispersible tablet formulation resulted in rapid and sustained improvements in hemoglobin. The study also showed that the drug led to a simultaneous reduction in hemolysis or destruction of red blood cells.

As a condition of accelerated approval, GBT will continue to investigate Oxbryta in the HOPE-KIDS 2 trial, a confirmatory post-approval study. GBT will continue to conduct the HOPE-KIDS 1 study to demonstrate the benefit of Oxbryta and potentially support its use in children from 9 months of age with SCD. The company is also conducting an ongoing open-label follow-up study for all patients under the age of 18 who have participated in Oxbryta clinical trials.

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