Following a successful appeal led by patients, the United Kingdom’s National Institute for Health and Care Excellence (NICE), has approved the use of Voxealtor in treating sickle cell disease across the country.
Last summer, UK health regulators initially rejected Voxealtor, impacting approximately 17,500 patients with sickle cell disease in the UK. This marks the first time that NICE has recommended a drug for sickle cell disease for routine use.
John James, Chief Executive of the Sickle Cell Society, welcomed the decision by the health agency, emphasizing its significant impact on patients’ lives.
James stated, “NICE’s decision brings hope and relief to many. We are deeply grateful for this milestone, which represents a transformative moment for those living with the condition.”
“Users of Voxealtor have shared remarkable stories of improved quality of life, reduced pain, and increased energy levels. This medication has enabled them to work, connect with loved ones, and enhance their overall well-being – things many of us often take for granted.”
“For too long, there has been inadequate investment in better care and effective treatments for sickle cell. Today, we celebrate this achievement and commend all those who contributed tirelessly to it.”
“Tomorrow, our advocacy continues as we strive for a future where new treatments for sickle cell are not a rare occurrence but a regular part of care for those living with the condition.”
It’s important to highlight that sickle cell disease disproportionately affects Black individuals, making this latest development a significant step towards ensuring equitable access to safe and effective treatments.
Despite being the fastest-growing genetic condition in the country, sickle cell disease has long been underserved in terms of treatment options.
An initial favorable assessment of Crizanlizumab for sickle cell patients was later reversed by regulators in an internal review, leaving many without a reliable treatment option.
Patients using Voxelator have reported remarkable improvements in their quality of life, citing reduced pain and increased energy levels.
Sickle cell disease gets its name from the abnormal transformation of healthy, oxygenated, O-shaped red blood cells into C-shaped cells due to abnormal hemoglobin proteins. These cells become hard and sticky, causing blockages in blood vessels, severe pain, and various complications such as delayed growth, strokes, and lung problems, necessitating frequent hospitalization.
Voxelator, taken orally, works by enhancing red blood cells’ ability to retain oxygen, thereby reducing the frequency of illness.
Hazel Attua, a sickle cell patient benefiting from Voxelator, praised the NHS for providing others with the opportunity to experience similar life-changing transformations.
“Being on Voxelator for nearly two years has truly transformed my life,” she said. “Since starting Voxelator, my energy has soared, making a massive change to my daily routine.
“This has not only improved my physical well-being but also my mental health, and I have a more positive outlook on life. I’m thrilled that others will have the opportunity to experience the same transformation and embrace life to the fullest.”
Susan Rienow, Country President at Pfizer UK, which helped develop the treatment, expressed delight at the positive NICE recommendation and interim funding through the Innovative Medicines Fund, providing eligible patients in England with immediate access.
