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Oxbryta Gets Marketing Authorisation In The UK For Patients With Sickle Cell Disease

Global Blood Therapeutics, Inc. announced on July 26 that the Medicines and Healthcare products Regulatory Agency (MHRA) has granted the UK marketing authorization for Oxbryta® (Voxelotor) for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in adult and paediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide (hydroxyurea).

Voxelotor, an oral treatment taken once daily, is the first medicine authorized in Great Britain that directly inhibits sickle haemoglobin (HbS) polymerization, the molecular basis of sickling and destruction of red blood cells in SCD.

The marketing authorization by the MHRA follows the European Commission (EC) authorization earlier this year, on February 16, 2022.

“We welcome the MHRA’s marketing authorization of Voxelotor as a new treatment option for many people in Great Britain living with sickle cell disease, a devastating life-long condition which for far too long has seen little therapeutic innovation,” said Beatriz F. Pujol, Ph.D., Vice President, Head of Medical Affairs EU & GCC at GBT.

“Following this marketing authorization by the MHRA, we look forward to working with the National Institute of Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) with the goal of helping to facilitate rapid access to Voxelotor for people living with sickle cell disease who may benefit from this important treatment.”

SCD affects approximately 15,000 people in the UK.1 People living with SCD experience progressive, serious complications and morbidities, including organ damage, which lead to decreased quality of life and early mortality. Furthermore, 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.

Results demonstrate clinically meaningful and statistically significant improvements in haemoglobin (Hb) levels, accompanied by a reduction of haemolysis markers, for patients treated with Voxelotor. 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 Voxelotor 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 haemolysis in indirect bilirubin and reticulocyte percentage.

In the HOPE Study, 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%).10

Results from the HOPE Study were published in June 2019 in The New England Journal of Medicine and the analysis of the complete data from the HOPE Study was published in The Lancet Haematology in April 2021.

  1. Sickle Cell Society. About Sickle Cell.
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