- Voxelotor is the first medicine in Britain to directly inhibit sickle haemoglobin polymerisation
- Global Blood Therapeutics (GBT) has announced that the Medicines and Healthcare products Regulatory Agency (MHRA) has granted Britain marketing authorisation for Oxbryta – also known as voxelotor.
It concerns the treatment of haemolytic anaemia due to sickle cell disease (SCD) in eligible adult and paediatric patients of 12 years and older, as monotherapy or in combination with hydroxycarbamide.
Oxbryta – an oral treatment taken once daily – is the first medicine authorised in Britain that directly inhibits sickle haemoglobin (HbS) polymerisation; the molecular basis of sickling and destruction of red blood cells in SCD.
- API – Voxelotor
- Originator-Global Blood Therapeutics
- Class-Antianaemics; Antifibrotics; Benzaldehydes; Ethers; Pyrazoles; Pyridines; Small molecules
- Mechanism of Action-Sickle haemoglobin inhibitors.
Voxelotor binds reversibly to hemoglobin, stabilizing the oxygenated hemoglobin state and preventing HbS polymerization by increasing hemoglobin’s affinity for oxygen.
- Orphan Drug Status-Yes – Sickle cell anaemia
- Indication – Sickle cell anaemia
Last year, Oxbryta was the first SCD treatment to receive a Promising Innovative Medicine designation from the MHRA, which subsequently granted the medicine a positive scientific opinion under the Early Access to Medicines Scheme. This enabled healthcare professionals to treat selected patients with Oxbryta prior to market authorisation based on clinical factors to address a clear unmet medical need.
“We welcome the MHRA’s marketing authorisation of voxelotor as a new treatment option for many people in Britain living with sickle cell disease, a devastating life-long condition which for far too long has seen little therapeutic innovation,” said Dr Beatriz Pujol, vice president at GBT.
“Following this marketing authorisation by the MHRA, we look forward to working with [the National Institute for Health and Care Excellence] NICE and the Scottish Medicines Consortium 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,” she added.
SCD affects approximately 15,000 people in the UK. People living with SCD experience progressive, serious complications and morbidities, including organ damage, which leads 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.
The marketing authorisation by the MHRA, which follows the European Commission authorisation earlier this year, is based on results demonstrating clinically meaningful and statistically significant improvements in haemoglobin levels, accompanied by a reduction of haemolysis markers.
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