Roche's Monthly Subcutaneous C5 Inhibitor PiaSky® (Crovalimab) Receives First EU Approval for PNH Treatment

On August 27, according to Roche's announcement, the European Commission has approved PiaSky® (crovalimab), a new generation C5 cyclic antibody, for the treatment of adult and adolescent (12 years and older, weighing 40 kg and above) patients with paroxysmal nocturnal hemoglobinuria (PNH) who have either not received or have been previously treated with a C5 inhibitor.

This also means that PiaSky has become the first monthly subcutaneous (SC) injection therapy for PNH in the EU, and patients can self-administer the injection after adequate training. This therapy may serve as an alternative to the current regular intravenous infusion of C5 inhibitor therapies, with the potential to help reduce the burden and life disruption for PNH patients and their caregivers.

Piasky is a recyclable humanized monoclonal antibody targeting complement protein C5, designed to block the complement system in the human immune system. Engineered through continuous monoclonal antibody recycling technology, its binding to C5 can induce the degradation of the C5 protein. Subsequently, this antibody can be released back into the extracellular space by binding to the FcRn receptor, allowing it to bind with other C5 proteins. As a result, Piasky can achieve rapid and sustained complement pathway inhibition at a lower dose.

The approval was based on the results of the Phase 3 COMMODORE 2 study, which showed that PiaSky, administered subcutaneously once every 4 weeks, provided good disease control and tolerability in PNH patients who had not previously received a C5 inhibitor. The efficacy of PiaSky was non-inferior to that of Eculizumab (eculizumab), a C5 inhibitor requiring intravenous administration once every 2 weeks. The incidence of adverse events in patients treated with PiaSky was similar to that of the active comparator drug (78% vs. 80%, respectively).

PNH is a rare and life-threatening syndrome, with patients suddenly presenting symptoms such as hematuria, anemia, and thrombosis. Inhibition of complement C5 is the standard treatment for PNH patients with significant clinical symptoms. However, the continuous complete suppression of the terminal complement pathway and the high serum concentration of complement C5 pose challenges for drug development, leaving patients with no choice but intravenous therapy. Eculizumab is currently the standard treatment for PNH.

Piasky is the first monthly subcutaneous injection treatment for PNH, and patients have the option to self-administer the drug outside of supervised medical facilities. According to an earlier press release from Roche, Piasky significantly improves both the efficacy and convenience of PNH treatment. In conventional blood transfusion therapy, PNH patients need frequent hospital visits for blood transfusions due to recurring hemolysis, with each transfusion session typically lasting 3-4 hours. In contrast, Piasky can be self-administered subcutaneously once every 4 weeks, offering greater convenience. Piasky has been approved in multiple regions worldwide, including China, the United States, and Japan.

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