Children with sickle cell disease (SCD) as young as age 4 could be prescribed Oxbryta (Voxelotor) if the U.S. Food and Drug Administration (FDA) approves a supplemental new drug application (sNDA) submitted by the therapy’s developers.
Global Blood Therapeutics (GBT) is asking the FDA to expand the use of Oxbryta — the first approved therapy targeting the underlying cause of SCD — to children ages 4 and older who have the inherited blood disease.
The therapy’s current label, allowing an oral daily dose of 1,500 mg, covers SCD patients ages 12 and older.
GBT also submitted a separate new drug application, or NDA, asking for the approval of a pediatric weight-based formulation of Oxbryta, which was found to result in similar blood-related benefits as those seen with the approved dose in older patients.
“Starting at a very early age, sickle cell disease has a serious and life-altering impact on children, and current therapeutic options for children under 12 years of age are limited,” Ted W. Love, MD, GBT’s President and CEO, said in a press release.
Both regulatory applications were proposed for priority review, which shortens the review process to six months from the standard 10 months. The FDA’s ruling on the completeness and acceptability of the applications, as well as on their potential for priority review, is expected within two months.
Love said GBT’s team “is committed to working closely with the FDA to potentially expand access to Oxbryta, thereby providing hope to families. We believe there is significant potential to impact the longer-term outcomes [of SCD] by addressing the root cause of red blood cell sickling at a young age.”
Oxbryta targets SCD’s underlying cause by increasing hemoglobin’s affinity to oxygen. That prevents its polymerization, or clumping, and the resulting red blood cell sickling and destruction. Haemoglobin is the protein inside red blood cells that is responsible for transporting oxygen. Through addressing haemolytic anemia and improving oxygen delivery throughout the body, GBT believes that Oxbryta has the potential to modify the course of SCD.
In recognition of the critical need for new SCD treatments, the FDA granted Oxbryta Breakthrough Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations for the treatment of patients with SCD. Additionally, Oxbryta has been granted Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), and the European Commission (EC) has designated Oxbryta as an orphan medicinal product for the treatment of patients with SCD. Also, in May 2021, Oxbryta was granted Promising Innovative Medicine (PIM) designation in the United Kingdom from the Medicines and Healthcare Products Regulatory Agency (MHRA).