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Global Blood Therapeutics’ treatment for patients with sickle cell disease approved in Europe

The once-daily oral drug is the first medicine approved in Europe to treat the condition in adults and paediatric patients 12 years and older

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Global Blood Therapeutics (GBT) has announced the European Commission’s (EC) decision to grant a marketing authorisation for Oxbryta (voxelotor) for the treatment of haemolytic anaemia caused by sickle cell disease (SCD) in adult and paediatric patients 12 years of age and older.

Under the authorisation, the treatment can be used as monotherapy or combined with hydroxycarbamide (hydroxyurea) and would be the first sickle haemoglobin polymerisation inhibitor to be approved in Europe. As a once-daily, oral treatment, Oxbryta works by increasing haemoglobin levels and reducing sickling and haemolysis.

Affecting approximately 52,000 people – primarily of Mediterranean, African and South Asian descent – SCD is one of the most prevalent genetic diseases in Europe. For those living with SCD, progressive and life-threatening complications – including damage to major organs – are common, contributing to decreased quality of life and early death.

Sebastian Stachowiak, head of Europe and GCC at GBT, said: “We are thankful to the investigators, patients and others in the sickle cell community who supported the development of this therapy and we hope it will make a very significant difference to the thousands of people in Europe living with the debilitating impact of this disease.”

The EC’s approval follows a positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) in December 2021 based on results of the phase 3 HOPE (Haemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) study.

The results, which were published in The New England Journal of Medicine in June 2021, showed statistically significant improvements in haemoglobin levels, accompanied by haemolysis for patients treated with Oxbryta. The analysis of the complete data from the HOPE study was published in The Lancet Haematology in April 2021.

Dr Baba Inusa, consultant and professor of paediatric haematology, Guy’s and St Thomas’ NHS Foundation Trust, London and chair of the National Haemoglobinopathy Panel in England, commented: “Treatment with Oxbryta has been shown to break the recurrent cycle of red blood cell sickling leading to their destruction – significantly improving patients’ haemoglobin levels and the blood’s oxygen-carrying capacity.”

With the EC’s approval, Oxybryta has received marketing authorisation in all EU member states, as well as in Iceland, Liechtenstein and Norway. GRT has submitted an application to the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for marketing authorisation using the EC Decision Reliance Procedure.

Article by
Fleur Jeffries

18th February 2022

From: Regulatory



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