Oxbryta recommended for NHS coverage to treat sickle cell disease
England's NICE draft guidance advises medicine for patients, 12 and up
England’s National Institute for Health and Care Excellence (NICE) has published final draft guidance recommending the country’s National Health Service (NHS) cover Pfizer’s Oxbryta (voxelotor) to treat sickle cell disease.
“Today’s approval by NICE marks a significant milestone in sickle cell care, addressing decades of limited access to disease-modifying treatments,” John James CEO of the Sickle Cell Society, said in a press release from Pfizer sent to Sickle Cell Disease News. “We applaud this decision and the work of everyone involved in making this day happen.”
The NICE final draft guidance recommends Oxbryta for hemolytic anemia, that is, low red blood cell counts due to red blood cell destruction, in sickle cell patients ages 12 and older. It may be used on its own or with the standard sickle cell treatment hydroxyurea, which is also called hydroxycarbamide, for those who can’t take hydroxyurea, or who have an inadequate response to it.
The move makes Oxbryta the first treatment for hemolytic anemia related to sickle cell to be recommended for NHS coverage by NICE, according to Pfizer. It’s expected to benefit up to 4,000 sickle cell patients in England.
“The NHS has worked hard to make this life-changing treatment available at a fair price for the taxpayer, as part of our wider drive to improve the quality and experience of care for sickle cell patients, and tackle the stigma and inequalities they have told me they face,” Amanda Pritchard, chief executive of the NHS, said in another press release.
“We are proud to make this new treatment available on the NHS — it could help thousands of patients living with sickle cell disease across the country to have a higher quality of life and experience fewer side effects, and represents a significant step forward in addressing the healthcare inequalities experienced by some of our communities,” added Bola Owolabi, director of the National Healthcare Inequalities Improvement Programme at NHS England.
What’s next for Oxbryta after recommendation?
The draft guidance will need to go through a consultation and is expected to be finalized in June. Until Oxbryta is available through the NHS, eligible patients can access the therapy thanks to funds from the Innovative Medicines Fund (IMF).
“We look forward to this being the first in a series of new treatment options for people living with sickle cell disorder,” James 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,” said Susan Rienow, president of Pfizer U.K.
Sickle cell is caused by mutations that result in an abnormal form of the oxygen-carrying protein hemoglobin being produced, which tends to form clumps in blood cells and deforming them into a sickle-like shape, resulting in their premature destruction.
Oxbryta binds to the defective hemoglobin and increases its affinity to bind to oxygen, making it less likely for it to clump up inside red blood cells. The therapy was originally developed by Global Blood Therapeutics, which was acquired by Pfizer in 2022. Oxbryta was approved in Great Britain ahead of the acquisition.
“We have remained committed to ensuring this treatment would reach eligible patients and we fully recognize the significance of this milestone for the sickle cell community. More broadly, we will continue to work with the community and other stakeholders to ensure we address the health inequalities that exist to help improve care and health outcomes,” Rienow said.
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