The European Medicines Agency (EMA) has accepted a marketing authorisation application for Global Blood Therapeutic’s (GBT) Oxbryta (voxelotor).
GBT is aiming for the approval of Oxbryta to treat haemolytic anaemia in patients with sickle cell disease (SCD) who are 12 years of age and older.
Oxbryta is a first-in-class oral therapy, designed to inhibit haemoglobin polymerisation, which causes the sickling and destruction of red blood cells in SCD.
This process – known as sickling – causes haemolytic anaemia and blockages in capillaries and small blood vessels.
In turn, this can impact the flow of blood and oxygen throughout the body, with reduced oxygen delivered to tissues and organs which can lead to life-threatening complications.
GBT’s EMA application is based on data from the Phase III HOPE study, as well as the Phase II HOPE-KIDS 1 Study.
The HOPE study achieved its primary endpoint of an improvement in haemoglobin (Hb) levels 0of greater than 1 g/dL at 24 weeks.
The EMA has already granted Oxbryta a Priority Medicines (PRIME) designation, with the European Commission having also designated Oxbryta as an orphan medicinal product for the treatment of patients with SCD.
“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,” said Ted Love, president and chief executive officer of GBT.
“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,” he added.