The Early Access to Medicines Scheme (EAMS) launched in 2014 and allows patients with unmet clinical needs to gain early access to unlicensed medicines and medicines to be used outside of their licence. The MHRA provides benefits and risk information on all medicines within the EAMS, and it is up to the discretion of the physician to understand these pros and cons and prescribe the EAMS medicine to their patient if they believe it to be an overall benefit.
Sickle Cell Disease (SCD) affects around 15,000 people in the UK today. It is a genetic condition in which red blood cells in the body contain an abnormal haemoglobin which leads red blood cells to form a ‘sickle’ or crescent shape. The general population have round red blood cells with and indent in the middle, much like a doughnut without a hole. The abnormal sickle shaped red blood cells have a lower affinity for oxygen, meaning that less oxygen is delivered to the muscles and organs compared to the conventional round-shaped red blood cells.
Complications which can occur in individuals with SCD are increased risk of stroke, blindness, bone damage, acute chest syndrome and damage to various organs over time.
Voxelotor is a treatment offered on the EAMS for SCD. It is a haemoglobin S polymerization inhibitor. In more simple terms, this prevents the red blood cells from forming the sickle/crescent shape. This allows individuals with SCD to produce more round red blood cells, and therefore deliver more oxygen to the various parts of the body.
Voxelotor has been studied in clinical trials of more than 800 SCD patients in over 25 studies. Its safety and effectiveness have been demonstrated on patients aged 12 and over.
Owing to the limited treatments currently available for SCD, and only one curative therapy offered in severe conditions (stem cell transplantation), Voxelotor has been implemented through the EAMS to give SCD patients more treatment options. This is especially important as some currently available treatments are not tolerated well by some SCD patients.
For more information, please refer to the links below:
Early access to medicines scheme applications: pending, refused, granted
Voxelotor in the treatment of sickle cell disease
https://www.gov.uk/government/publications/voxelotor-in-the-treatment-of-sickle-cell-disease
What is Sickle Cell Disease?