Pfizer’s Oxbryta (voxelotor) has been recommended by the National Institute for Health and Care Excellence (NICE) to treat sickle cell disease (SCD) in patients aged 12 years and older.
Affecting around 17,000 people in England, SCD is a life-long genetic disorder that causes red blood cells to take a distinct crescent shape. This can block blood vessels and affect the way oxygen is carried around the body.
The disease can cause serious health problems, including haemolytic anaemia – a blood condition that occurs when red blood cells are destroyed faster than they are replaced – as well as severe organ damage, intense pain, strokes and sight loss.
Pfizer’s Oxbryta, taken as a once-daily tablet, was shown in clinical trials to improve anaemia compared to standard care and demonstrated the potential to reduce the need for repeated blood transfusions.
The drug can be used with or without another SCD treatment called hydroxycarbamide, according to NICE’s final draft guidance, and is expected to benefit about 4,000 people with SCD in England straight away.
The agency’s approval comes after a reduction in price “mitigated some of the uncertainties” in the evidence identified by an independent appraisal committee in its previous draft guidance and means the treatment, which will be funded immediately through NHS England’s Innovative Medicines Fund, can now be recommended as a cost-effective use of the health service’s resources.
Susan Rienow, country president for Pfizer UK, said: “We are delighted with this positive NICE recommendation and interim funding through the Innovative Medicines Fund, which will give eligible patients in England immediate access.
“We have remained committed to ensuring this treatment would reach eligible patients and we fully recognise the significance of this milestone for the sickle cell community.”
Pfizer gained access to Oxbryta in October 2022 through its $5.4bn acquisition of Global Blood Therapeutics.
Commenting on the latest authorisation for the therapy, Helen Knight, director of medicines evaluation at NICE, said: “This is great news for people with SCD, particularly given the health inequalities experienced by people with the condition.
“Throughout our appraisal of voxelotor, the appraisal committee has always considered that it has the potential to address the need for effective treatments for SCD…”