EC Approves Roche’s PiaSky for PNH Treatment

The European Commission (EC) has granted approval for Roche’s new monoclonal antibody, PiaSky (crovalimab), designed for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) in both adults and adolescents. This groundbreaking therapy, which inhibits the complement protein C5, stands out as the first monthly subcutaneous treatment for PNH to receive approval in the European Union.

This approval opens a new avenue for patients aged 12 and above who weigh at least 40kg, presenting them with a less invasive treatment option. PiaSky’s regulatory green light is predominantly supported by the results from the Phase III COMMODORE 2 study, which focused on patients who had not been previously treated with C5 inhibitors. The study, which was randomised and open-label, compared the effectiveness and safety of PiaSky against eculizumab in PNH patients.

The findings showed that PiaSky, administered through subcutaneous injections every four weeks, provided disease control comparable to that of eculizumab. Furthermore, the safety profile of PiaSky was confirmed to be on par with eculizumab, showing similar rates of adverse events among the patients. The approval is also backed by data from two additional Phase III studies, COMMODORE 1 and COMMODORE 3, which further reinforced the application.

PiaSky’s appeal lies in its ability to offer a monthly treatment option that can be self-administered after proper training, making it less cumbersome for patients. This convenience is a significant advancement for those living with PNH, as it reduces the frequency of medical visits and the overall treatment burden. PiaSky has already been approved in multiple regions, including the United States and Japan, based on comprehensive evidence from the COMMODORE clinical trials.

Roche is committed to further exploring the potential of PiaSky through a robust clinical development program that includes five Phase III studies and three earlier phase trials. These studies aim to investigate PiaSky’s efficacy in treating various complement-mediated diseases such as PNH, atypical haemolytic uremic syndrome, and sickle cell disease.

Levi Garraway, Roche’s global product development head and chief medical officer, expressed optimism about the new treatment option. He highlighted that PiaSky combines effective disease control via C5 inhibition with innovative recycling technology, allowing for its monthly subcutaneous administration. This new treatment option is expected to significantly alleviate the treatment burden for many individuals living with PNH in Europe.

In addition to its recent clinical achievements, Roche reported a net income of SFr6.6bn ($7.4bn) in the first half of 2024, which marks a 4% decline at constant exchange rates compared to the SFr7.5bn recorded in the same period of 2023.