MHRA grants marketing authorisation to Global Blood Therapeutics for sickle cell disease drug


The Medicines and Healthcare products Regulatory Agency (MHRA) has granted Global Blood Therapeutics (GBT) a British marketing authorisation for Oxbryta – also known as voxelotor – for both adult and paediatric patients 12 years of age and older requiring treatment of haemolytic anaemia due to sickle cell disease (SCD).

The authorisation supports the use of Oxbryta as either a monotherapy or for use in combination with hydroxycarbamide, also known as hydroxyurea.

Oxbryta is an oral treatment taken once daily and is the first medicine authorised in Britain that directly blocks sickle haemoglobin (HbS) polymerisation, which is the molecular foundation of sickling and the destruction of red blood cells in SCD.

In the UK, approximately 15,000 people are affected by SCD, a progressive and complex condition which can cause serious complications, including organ damage. For those living with the condition, it is common to experience economic disadvantages and health inequalities as SCD can inflict negative societal impacts in areas like healthcare, education and employment.

Beginning in early childhood, SCD complications can include neurocognitive impairment, acute chest syndrome and silent and overt stroke. It has been shown that early intervention and treatment of SCD can potentially change the course of this disease and, in turn, reduce symptoms and events while preventing long-term organ damage and extending life expectancy.

The marketing authorisation from the MHRA follows the European Commission (EC) authorisation which was made earlier this year and is based on data taken from the phase 3 HOPE trial. The results demonstrated clinically meaningful and statistically significant improvements in haemoglobin (Hb) levels, accompanied by a reduction of haemolysis markers, for patients treated with Oxbryta.

In 2021, Oxbryta was the first SCD treatment to be granted a Promising Innovative Medicine (PIM) designation from the MHRA, which then allowed the medicine to receive a positive scientific opinion under the Early Access to Medicines Scheme (EAMS). Healthcare professionals were then able to treat selected patients with Oxbryta ahead of market authorisation, based on clinical factors to address a clear unmet medical need.

Dr Beatriz Pujol, vice president, head of medical affairs EU & GCC at GBT, said: “Following this marketing authorisation 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.”



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