VOXELOTOR
May 2022
NHP Voxelotor Guideline published
Voxelotor is a new therapy indicated for the treatment of haemolytic anaemia in adult and paediatric patients 12 years and older with sickle cell disease (SCD). Voxelotor is an orally administered hemoglobin S polymerization, inhibitor which increases the affinity of HbS for oxygen, helping red blood cells deliver oxygen throughout the body. It can be administered alone or in combination with hydroxycarbamide.
In January 2022 Voxelotor was given a positive Scientific opinion for the Early Access to Medicine Scheme (EAMS), which programme allows for promising new unlicensed medicines to be used outside their licence, to UK patients that have a high unmet clinical need.
The NHP Voxelotor Working Group and general panel, have published a guideline for the use and administration of this drug.
NB: Linked document updated September 2022