Why carry out this study?

The efficacy of ravulizumab (intravenous [IV] formulation; administered every 8 weeks) for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) has been demonstrated in randomized trials.

This ongoing pivotal phase 3 trial was designed to compare the pharmacokinetic (PK) non-inferiority of the ravulizumab on-body delivery system for subcutaneous (SUBQ) administration (self-administered weekly) with IV ravulizumab in patients with PNH who were clinically stable on prior IV eculizumab therapy.

What was learned from the study?

In patients with PNH, treatment with SUBQ ravulizumab achieved PK non-inferiority compared with IV ravulizumab for the primary endpoint of day 71 serum ravulizumab trough concentration (Ctrough).

Through 1 year of SUBQ ravulizumab treatment, ravulizumab Ctrough levels were maintained above the previously established PK threshold and free complement component 5 (C5) levels below the defined pharmacodynamic threshold; all efficacy endpoints remained stable over time, and patients reported increased satisfaction with SUBQ ravulizumab (compared with IV eculizumab).

Patients may be switched from IV eculizumab or IV ravulizumab to SUBQ ravulizumab without loss of efficacy, demonstrating that the SUBQ route of administration provides an additional treatment choice for patients with PNH receiving complement C5 inhibitor therapy.