Iptacopan (3) – Fabhalta®

• Clinical trials
  • The APPLY-PNH trial is a completed, multicentre, open-label RCT comparing iptacopan with eculizumab or ravulizumab.

a) Adults with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia and have not received prior treatment

• Additional benefit is not proven for iptacopan as monotherapy.

b) Adults with paroxysmal nocturnal haemoglobinuria (PNH) who continue to have haemolytic anaemia and have received prior treatment

• No additional benefit of Iptacopan as monotherapy is not proven for patient group b).
• For patient group b), no suitable data are available for comparing Iptacopan with the appropriate comparator therapy, as the APPLY-PNH study did not involve a switch to pegcetacoplan or danicopan in combination with eculizumab or ravulizumab.
• Irrespective of the failure to implement the appropriate comparator therapy, it cannot be assumed with the necessary certainty, against this background, that the analyses submitted by the pharmaceutical manufacturer or sensitivity analyses, with regard to the assumptions made in defining the analysis populations, would in fact be suitable for identifying patients for whom a switch to proximal complement inhibition is not yet indicated.

Courtesy translation only, please refer to the German original.