GBT Announces Plans to Seek Regulatory Approval for Oxbryta® (Voxelotor) to Treat Sickle Cell Patients in Europe
Global Blood Therapeutics (GBT) plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Oxbryta (Voxelotor) be approved for treating hemolytic anemia in patients with sickle cell disease (SCD) who are 12 or older. The company plans to submit the MAA by mid-2021.
“Currently, there are no approved therapies in Europe to treat hemolytic anemia in sickle cell disease,” Ted W. Love, MD, GBT president and CEO, stated in the company’s press release. “We hope to address this critical unmet need as part of our commitment to patient access to innovative treatments for this devastating disease.”
In Europe, an estimated 52,000 people are living with SCD. In recognition of the need for new SCD treatments, the EMA has already granted Oxbryta Priority Medicines (PRIME) designation, and the European Commission (EC) designated Oxbryta as an orphan medicinal product for the treatment of patients with SCD. Additionally, on November 25, 2019, Oxbryta received U.S. Food and Drug Administration (FDA) accelerated approval for the treatment of SCD in adults and children 12 years of age and older.
About Oxbryta® (Voxelotor) Tablets
Oxbryta (Voxelotor) is an oral, once-daily therapy for patients with sickle cell disease (SCD). Oxbryta works by increasing haemoglobin’s affinity for oxygen. Since oxygenated sickle haemoglobin does not polymerize, GBT believes Oxbryta blocks polymerization and the resultant sickling and destruction of red blood cells, which are primary pathologies faced by every single person living with SCD. With the potential to improve haemolytic anemia and oxygen delivery, GBT believes that Oxbryta has the potential to modify the course of SCD.
“Anemia and hemolysis are well established to cause morbidity and mortality in sickle cell disease. At GBT, our focus is to address sickle cell disease at its core and modify the course of the disease, with the goal of mitigating the serious and life-threatening complications that often lead to long-term damage and early death,” Love said.
Results from HOPE trial, which compared Oxbryta to a placebo as a once-daily oral treatment for six months, found robust and sustained improvements in the amount of healthy, circulating red blood cells in teenagers and adults. The approved 1,500 mg dose raised haemoglobin levels in 51% of patients, compared with 6.5% for those who took a placebo. Biomarkers of red blood cell damage also were lowered with this high dose: reticulocytes (by 19.9%) and bilirubin (by 29.1%).
Read GBT’s Full Press Release on Oxbryta HERE
More information on Oxbryta is available HERE
