MHRA grant marketing authorisation for sickle cell anaemia treatment

pharmafile | July 26, 2022 | News story | Manufacturing and Production  

Global Blood Therapeutics (GBT) has announced the MHRA marketing authorisation for Oxbryta® (voxelotor) for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in eligible adult and paediatric patients aged 12 years and older.

The treatment has received a marketing authorisation as a monotherapy or in combination with hydroxycarbamide (hydroxyurea).

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

“We welcome the MHRA’s marketing authorisation 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 Dr Beatriz F Pujol, vice president, head of medical affairs EU & GCC at GBT.

SCD affects around 15,000 people in the UK, and those living with the disease experience progressive, serious complications and morbidities. An estimated 100,000 in the US live with the disease, alongside a further 52,000 in Europe, and up to 100,000 in Brazil.

Complications for the disease include organ damage, leading 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.

SCD is a lifelong inherited blood disorder that impacts haemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body.

Dr Beatriz continued: “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.”

Ana Ovey

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