1 Recommendations

1.1 Ravulizumab is recommended, within its marketing authorisation, as an option for treating paroxysmal nocturnal haemoglobinuria in adults:

  • with haemolysis with clinical symptoms suggesting high disease activity, or

  • whose disease is clinically stable after having eculizumab for at least 6¬†months, and

  • the company provides it according to the commercial arrangement.

Why the committee made these recommendations

Paroxysmal nocturnal haemoglobinuria is currently treated with eculizumab infusions every 2 weeks.

Clinical trial evidence shows that ravulizumab is similarly as effective as eculizumab and is just as safe. Ravulizumab is given less often than eculizumab so there is some benefit on quality of life. Also, it may save costs because people need to have it less often.

Ravulizumab is as effective and costs less than eculizumab so it is recommended as an option for treating paroxysmal nocturnal haemoglobinuria.

  • National Institute for Health and Care Excellence (NICE)