SCD Drug Oxbryta Receives CHMP Positive Opinion For Approval In The EU
If approved by the European Commission, Oxbryta would be the first medicine available in Europe that directly inhibits sickle hemoglobin (HbS) polymerization, the molecular cause of sickling and destruction of red blood cells in SCD.
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization for Oxbryta® (voxelotor) tablets for the treatment of hemolytic anemia due to sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide (hydroxyurea).
Based on this CHMP opinion, a decision by the European Commission, which authorizes marketing approval in the European Union, is expected in the first quarter of 2022. If approved by the EC, Oxbryta will receive marketing authorization in all EU member states and Iceland, Liechtenstein and Norway.
“For far too long, people battling the devastating effects of sickle cell disease have had few therapeutic options. This positive CHMP opinion marks an important step forward to a new medicine for tens of thousands of people in Europe living with the disease,” said Ted W. Love, M.D., President and Chief Executive Officer of Global Blood Therapeutics, the company behind Oxbryta’s development.
“In clinical studies, voxelotor has demonstrated significant increases in hemoglobin levels and reductions in markers of hemolysis, which is expected to improve quality of life and, we hope, to reduce chronic organ damage associated with the disease,” said Professor Mariane de Montalembert, Co-head of the Necker Site for major sickle cell syndromes and other rare pathologies of red blood cells and erythropoiesis, Necker-Enfants Malades Hospital, Sickle Cell Center in Paris.
Oxbryta is currently approved in the United States for the treatment of SCD in adults and children 12 years of age and older. On December 17, 2021, the U.S. Food and Drug Administration (FDA) has agreed to expand the use of Oxbryta to children as young as 4 years old. In addition, the Ministry of Health and Prevention (MOHAP) in the United Arab Emirates (UAE) has granted marketing authorization for Oxbryta for the treatment of SCD in adults and children 12 years of age and older.
How does Oxbryta work?
Oxbryta (voxelotor) is an oral, once-daily therapy for patients with sickle cell disease (SCD). Oxbryta works by increasing hemoglobin’s affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, Oxbryta inhibits sickle hemoglobin polymerization and the resultant sickling and destruction of red blood cells leading to hemolysis and hemolytic anemia, which are primary pathologies faced by every single person living with SCD. Through addressing hemolytic anemia and improving oxygen delivery throughout the body, GBT believes that Oxbryta has the potential to modify the course of SCD. In November 2019, the U.S. Food and Drug Administration (FDA) granted accelerated approval for Oxbryta tablets for the treatment of SCD in adults and children 12 years of age and older.12
As a condition of accelerated approval in the United States, GBT will continue to study Oxbryta in the HOPE-KIDS 2 Study, a post-approval confirmatory study using transcranial Doppler (TCD) flow velocity to assess the ability of the therapy to decrease stroke risk in children 2 to 14 years of age.
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 was granted Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), Oxbryta was designated by the European Commission (EC) as an orphan medicinal product for the treatment of patients with SCD, and Oxbryta was granted Promising Innovative Medicine (PIM) designation in the United Kingdom from the Medicines and Healthcare products Regulatory Agency (MHRA).
Where is the positive CHMP decision based on?
The CHMP opinion is based on data demonstrating clinically meaningful and statistically significant improvements in hemoglobin (Hb) levels, accompanied by reductions in red blood cell destruction (hemolysis), for patients treated with Oxbryta. Data from the Phase 3 HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study of 274 patients 12 years of age and older with SCD showed that, after 24 weeks of treatment, 51.1% of patients receiving Oxbryta achieved a greater than 1 g/dL increase in Hb compared with 6.5% receiving placebo (p<0.001), with significant improvements in markers of hemolysis in indirect bilirubin and reticulocyte percentage.9 Results from the HOPE Study were published in June 2019 in The New England Journal of Medicine. Oxbryta showed a favorable safety profile with limited and transitory adverse reactions. The most common adverse reactions occurring in ≥10% of patients treated with Oxbryta with a difference of >3% compared to placebo were headache (26% vs. 22%), diarrhea (20% vs. 10%), abdominal pain (19% vs. 13%), nausea (17% vs. 10%), fatigue (14% vs. 10%), rash (14% vs. 10%) and pyrexia (12% vs. 7%)
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