pharmafile | January 28, 2021 | News story | | Global Blood Therapeutics
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The EMA has accepted Global Blood Therapeutics (GBT)’s Marketing Authorisation Application (MAA) for Oxbryta (voxelotor) tablets, and has started its standard review process.
GBT is seeking full marketing approval from the EMA for Oxbryta to treat haemolytic anaemia in patients with sickle cell disease (SCD) who are 12 years of age and older.
A first-in-class oral, once-daily therapy, Oxbryta directly inhibits haemoglobin polymerisation, the root cause of the sickling and destruction of red blood cells in SCD. The sickling process causes haemolytic anaemia (low haemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.
The EMA’s acceptance is based on data from the Phase III HOPE study and the Phase II HOPE-KIDS 1 study, both of which enrolled patients at clinical sites in Europe. The HOPE study achieved its primary endpoint of an improvement in haemoglobin (Hb) levels of greater than 1g/dL at 24 weeks with significant improvements in markers of haemolysis in indirect bilirubin and reticulocyte percentage.
Analysis of the complete data from the HOPE study further demonstrated that Oxbryta, at a daily dose of 1500mg, resulted in durable improvements in Hb levels and markers of haemolysis over 72 weeks of treatment in SCD patients 12 years of age and older. Treatment with the drug was well tolerated, with no new safety or tolerability issues identified.
The EMA previously granted Oxbryta Priority Medicines (PRIME) designation, and the European Commission designated the drug as an orphan medicinal product for the treatment of patients with SCD.
Dr Ted W Love, President and CEO of GBT, said: “Sickle cell disease has a devastating impact on the lives of patients and their families, including serious and life-threatening complications that can lead to organ damage and early death.
“Despite this overwhelming need, there are currently no approved therapies in Europe that have the potential to modify the course of the disease. We look forward to working with the EMA to meet our goal of bringing the first treatment for haemolytic anaemia in sickle cell disease to European patients as soon as possible.”
Oxbryta is approved in the US for the treatment of SCD in adults and children 12 years of age and older.
Darcy Jimenez