Positive CHMP opinion for sickle cell disease anaemia tablet

pharmafile | December 20, 2021 | News story | Sales and Marketing  

Global Blood Therapeutics (GBT) have announced the CHMP adopted a positive opinion recommending marketing authorisation for Oxbryta®  (vocelotor) tablets. Oxbryta is for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in adults and paediatric patients 12 years of age and older, as a monotherapy or in combination with hydroxycarbamide (hydroxyurea).

People with sickle cell disease produce unusually shaped red blood cells. These cells can block blood vessels, and do not live as long as healthy blood cells. Sickle cell disease is a lifelong health condition, though treatment can help manage many of the symptoms. Those who suffer SCD experience anaemia, increased risk of serious infections, and painful episodes called sickle cell crises, which can be very severe, and last up to a week. Patients also experience progressive, serious complications and morbidities, including end-organ damage, which lead to decreased quality of life and early mortality. Economic disadvantages and health inequalities experienced by many patients with SCD can have negative societal impacts in areas such as access to healthcare, education, and employment. 

If approved by the EC, Oxbryta will receive marketing authorisation in all EU member states  as well as Iceland, Liechtenstein, and Norway. Oxbryta is an oral treatment taken once daily. If approved, the tablets would be the first medicine available in Europe that directly inhibits sickle hemoglobin (HbS) polymerisation, the molecular cause of sickling and destruction of red blood cells in SCD.

“The positive CHMP decision is of huge significance for the SCD community, and as a clinician, I would welcome the availability of this new therapeutic approach for my patients,” 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. “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.”

Ana Ovey

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