Benzinga | Dec 17, 2021 02:09PM EST

Global Blood Therapeutics Confirms Earlier Report FDA Approves Supplemental New Drug Application For Expanded Indication Of Oxbryta For Children As Young As 4 Years Of Age With Sickle Cell Disease

Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced the U.S. Food and Drug Administration (FDA) has granted accelerated approval of a supplemental New Drug Application (sNDA) for Oxbryta(r) (voxelotor) tablets for the treatment of sickle cell disease (SCD) in children ages 4 to less than 12 years. This approval expands the previously approved use of Oxbryta to treat SCD in patients ages 12 years and older in the United States. The FDA also approved GBT's separate New Drug Application (NDA) for Oxbryta (voxelotor) tablets for oral suspension, a new dispersible, once-daily tablet dosage form suitable for patients ages 4 to less than 12 years as well as for older patients who have difficulty swallowing whole tablets. Oxbryta is the first and only approved medicine that directly targets sickle hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD.

"For decades, the sickle cell disease community has been profoundly underserved, and there have been limited treatment options for younger patients with their whole lives ahead of them. Complications of SCD that can cause irreversible organ damage are known to begin in the first few years of life, which is why earlier intervention is critical," said Ted W. Love, M.D., president and chief executive officer of GBT. "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."

Of the more than 100,000 people in the United States living with SCD,1 an inherited blood disorder that causes lifelong health challenges, approximately 16,000 are children between the ages of 4 to 11 years.2 Complications of SCD begin in early childhood and include neurocognitive impairment, acute chest syndrome, and silent and overt stroke, among other serious issues.3 In addition, SCD limits children's educational attainment and social lives due to disease complications that often lead to repeated hospitalizations.4-6 Early intervention and treatment of SCD are critical and have shown potential to modify the course of this disease, reduce symptoms and events, prevent long-term organ damage and extend life expectancy.7

Oxbryta will now be available in two dosage forms for patients 4 years and older based on the patient's age, weight, and ability to swallow tablets: 500 mg tablets and 300 mg dispersible tablets. The dispersible tablet form includes grape flavoring and is intended to be dispersed in room-temperature clear drinks for ease of swallowing (such as drinking water or clear soda), making Oxbryta easier to take for patients who have difficulty swallowing whole tablets, particularly younger children.

The FDA's approval of the sNDA is based on data from the open-label Phase 2a HOPE-KIDS 1 Study (GBT440-007), which showed that weight-based treatment with the dispersible tablet formulation of Oxbryta resulted in rapid and sustained improvements in hemoglobin. Concurrent reduction of hemolysis (or red blood cell destruction) was also demonstrated.8

"As clinicians, we have had very few approved treatment options for sickle cell disease and many patients are untreated, which is very troubling for younger children who may benefit from early intervention," said Clark Brown, M.D., Ph.D., director of sickle cell clinical research at the Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta and a primary investigator of the Phase 2a HOPE-KIDS 1 Study. "This approval is a significant milestone in the treatment of younger children with SCD -- providing a therapy that addresses the root cause of the disease. I believe the new dispersible tablets can help patients and their caregivers achieve daily beneficial treatment and that Oxbryta will make a meaningful difference in improving the lives of children living with sickle cell disease."

"The Sickle Cell Disease Association of America congratulates GBT for the approval of Oxbryta in children as young as 4 years old. Having the new formulation and approval down to 4 years of age is meaningful for families living with sickle cell disease and the community more broadly," said Lewis Hsu, M.D., Ph.D., chief medical officer of the Sickle Cell Disease Association of America. "Due to the many medical complications associated with sickle cell disease, kids face challenges every day in their ability to go to school and have a typical childhood. Adding another new FDA-approved treatment option for children will enhance care and provide more choices for children living with sickle cell disease. SCDAA will advocate for patient-centered decision aids to guide choices. We will work closely with GBT, our partners, and the sickle cell community to promote awareness and education about this new therapy to allow patients to make informed decisions, while advocating for equitable access to care."

The FDA instituted its accelerated approval pathway to allow for earlier approval of drugs that treat serious conditions and that fill an unmet medical need based on a surrogate endpoint.9 As a condition of accelerated approval, GBT will continue to study Oxbryta in the HOPE-KIDS 2 Study, a post-approval confirmatory study using transcranial Doppler flow velocity to assess the ability of the therapy to decrease stroke risk in children 2 to 14 years of age. In addition, GBT is continuing to conduct the HOPE-KIDS 1 Study to demonstrate the benefit of Oxbryta and support its potential use in children with SCD as young as 9 months of age, and an ongoing open-label extension study for all patients less than 18 years of age who have participated in Oxbryta clinical trials.

GBT is committed to ensuring that patients who are prescribed Oxbryta can access their medicine. GBT Source Solutions(r) was established by GBT as a comprehensive program for patients who are prescribed Oxbryta that provides a wide range of practical, educational and financial support customized to each patient's needs. GBT Source Solutions provides support by reviewing insurance coverage options and explaining benefits; explaining specialty pharmacy benefits and working with the specialty pharmacies that coordinate shipments of Oxbryta; helping eligible, commercially insured patients with co-pay assistance; and helping appropriate patients stay on treatment with a nurse support team. More information is available at Oxbryta.com or 1-833-428-4968 (1-833-GBT-4YOU).

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