Weekly Self-Administered ULTOMIRIS Drug Shows Equivalent Performance to IV Treatment in Phase 3 Trial

Alexion Pharmaceuticals has delivered promising news from a pivotal Phase 3 study evaluating a subcutaneous formulation of ULTOMIRIS (ravulizumab-cwvz), a leading complement inhibitor for rare blood disorders. The trial successfully met its primary endpoint, demonstrating that the innovative ultomiris drug delivered via injection performed comparably to the traditional intravenous version.

What the Data Shows

The randomized study enrolled 136 patients with paroxysmal nocturnal hemoglobinuria (PNH) who had previously stabilized on complement therapy. Participants were split 2:1 between the new subcutaneous ULTOMIRIS drug and the established IV treatment. By Day 71, the subcutaneous version achieved non-inferiority in serum trough concentrations (p < 0.0001), meaning patients received consistent, effective complement inhibition without sacrificing efficacy.

Key findings included stable lactate dehydrogenase levels and maintained suppression of free C5 protein across all patients. Safety data remained consistent with the known profile of this ultomiris drug formulation, with no unexpected adverse events or serious infections reported during the 71-day treatment period.

A Game-Changer for Patient Convenience

The breakthrough potential lies in delivery. Each weekly dose uses specifically designed, patient-friendly devices that adhere to the skin and can be self-administered with a simple button push. Patients can complete their full dose hands-free in approximately 10 minutes—a significant departure from the time commitment of intravenous infusions.

Out of 135 patients who completed the controlled phase, all but one elected to continue with the subcutaneous ultomiris drug in the ongoing extension study, suggesting strong real-world preference for this treatment option.

Road to Approval and Beyond

Alexion now anticipates filing for regulatory approval in the U.S. and European Union during Q3 2021 for the subcutaneous ULTOMIRIS drug and device combination. The submission will cover two indications: PNH and atypical hemolytic uremic syndrome (aHUS).

If approved, this version could become the first subcutaneous complement inhibitor option for both conditions, potentially reshaping treatment patterns for patients managing these devastating ultra-rare disorders. The self-administration capability may improve quality of life by reducing hospital visits and offering greater autonomy over treatment schedules.

The Clinical Significance

For physicians treating PNH and aHUS, the ultomiris drug formulation represents a meaningful advance. Patients with these conditions face serious complications including hemolysis and blood clots. Having a subcutaneous option that maintains the same complement inhibition profiles as the IV version expands treatment accessibility while preserving clinical efficacy.

The company plans to gather 12 months of safety data from the extension period before final submissions, ensuring comprehensive evidence of long-term safety and tolerability for regulatory authorities worldwide.