Sickle cell disease (SCD) new drug! The first drug to treat the root causes of the disease, Oxbryta (voxelotor), is under EU review and is on the market in the US!

Global Blood Therapeutics (GBT) recently received a marketing authorization application (MAA) from Oxbryta (VOXelotor) from the European Medicines Agency (EMA) and has initiated a standard review process.
maa sought full approval from the EMA for Oxbryta to treat hemolytic anemia in patients aged 12 years and older with sickle cell disease (SCD).
, the EMA has granted voxelotor priority drug eligibility (PRIME) and orphan drug eligibility for SCD treatment.
U.S. regulatory aspects, Oxbryta received FDA accelerated approval in November 2019 to treat hemolytic anemia in ≥ 12-year-old SCD children and adult patients.
was approved through the FDA's Priority Review Channel, which took just over two months from the time the new drug application (NDA) was processed to the time it was finally approved.
, the FDA has granted voxelotor breakthrough drug eligibility (BTD), fast-track status, orphan drug eligibility, and rare pediatric diseases.
As a condition of FDA accelerated approval, GBT will continue to investigate Oxbryta in the HOPE-KIDS 2 study, a post-approval validation study that uses transcranial Doppler (TCD) blood flow velocity to assess Oxbryta's ability to reduce stroke risk in children ages 2-15.
Oxbryta is the first (first-in-class), once-a-day, oral drug, and the first approved treatment to directly inhibit sickle hemoglobin polymerization.
hemoglobin polymerization is the underlying cause of SCD red blood cell sickle and destruction.
sickle process can lead to hemolytic anemia (red blood cell damage leads to lower hemoglobin levels), blockages of capillaries and small blood vessels, and hinders the flow of blood and oxygen throughout the body.
reduction in oxygen supply to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage Oxbryta's active drug becomes voxelotor (formerly known as GBT440), which works by increasing hemoglobin's affinity for oxygen.
because oxygenated sickle hemoglobin does not converg, voxelotor blocks polymerization and the resulting sickle and destruction of red blood cells.
voxelotor improves hemolysis anemia and oxygen transport and potentially alters the process of SCD. Ted W. Love, President and CEO of
GBT, said, "Sickle cell disease (SCD) can have devastating effects on the lives of patients and their families, including serious life-threatening complications that can lead to organ damage and premature death.
, despite the huge demand, there is currently no approved treatment in Europe that has the potential to change the course of the disease.
look forward to working with EMA to bring the first treatment for SCD hemolysmolysis to patients in Europe as soon as possible.
"voxelotor molecular structure (Photo: drugapprovalsint.com) In Europe, Oxbryta's Marketing Authorization Application (MAA) is based on data from phase 3 HOPE studies and Phase 2 HOPE-KIDS 1 studies, both of which included patients at clinical trial sites in Europe.
HOPE-KIDS 1 is an open-label, single-dose, and multi-dose study that evaluates the safety, tolerance, pharmacodynamics, and exploratory efficacy of voxelotor for the treatment of SCD pediatric patients (4-17 years of age).
HOPE is a randomized, double-blind, placebo-controlled Phase 3 study conducted in 60 research institutions in 22 countries around the world that assessed the efficacy and safety of two dose levels of voxelotor (1500mg and 900mg, once a day oral) and placebo treatment for SCD.
HOPE study included 274 patients aged 12 and over with SCD, the majority of whom suffered from sickle cell anemia (pure hemoglobin S or hemoglobin S beta0-thalassemia), with about two-thirds of the baseline being treated with hydroxyurea.
study, patients were randomly grouped at 1:1:1 and received 1,500 mg voxelotor, 900 mg voxelotor, or placebo once a day.
the main endpoint is the proportion of patients who achieve hemoglobin response in intentional therapy analysis, defined as an increase of more than 1.0g/L in hemoglobin relative to baseline in week 24.