We are relieved and delighted at today’s news that The National Institute for Health and Care Excellence (NICE) have approved Voxelotor (Oxbryta) for use in people living with sickle cell disorder (SCD). This drug has transformed the way people living with the condition are able to live their lives, bringing improvements in physical and mental health that are so often denied to them.

Despite being the fastest-growing genetic condition in the UK, sickle cell disorder has historically been underserved by treatment options. Over the past two decades, only one licensed treatment has been available, and patients have experienced substandard care and negative attitudes from healthcare staff when accessing treatment.

Voxelotor, a drug initially rejected by NICE in July 2023, has now received this crucial approval following a successful appeal by the Sickle Cell Society and Pfizer. This decision not only signifies a triumph for the 17,500 individuals living with sickle cell disorder in the UK, but also represents a landmark victory in the fight for equitable access to safe and effective treatments.

Users of Voxelotor have reported remarkable improvements in quality of life, including reduced pain and increased energy levels, enabling them to reclaim aspects of life previously thought unattainable.

Voxelotor will be funded immediately via NHS England’s Innovative Medicines Fund. More from NICE in their news article here