Ravulizumab (Ultomiris®) is recommended as an option for use within NHS Wales for the treatment of paroxysmal nocturnal haemoglobinuria in paediatric patients with a body weight of 10 kg or above:

• in patients with haemolysis with clinical symptom(s) indicative of high disease activity
• in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months

This recommendation applies only in circumstances where the approved Patient Access Scheme (PAS) is utilised or where the list/contract price is equivalent or lower than the PAS price.